Materiały źródłowe

• #1 Neurofibromatosis type 2 (NF2)

  https://www.nhs.uk/conditions/neurofibromatosis-type-2/treatment/

  Theres currently no cure for neurofibromatosis type 2 (NF2), but there are treatments that can help manage your symptoms. […] Depending on your symptoms, treatments for NF2 include: hearing aids or cochlear implants (a cochlear implant is a small device that stimulates the nerve that sends sound signals to your brain), medicine that can stop tumours growing and sometimes improve hearing, surgery to remove tumours that are at risk of causing problems, using a beam of radiation to treat small tumours or pieces of a larger tumour that are left after surgery (radiotherapy). […] Most people with NF2 will need surgery at some point during their life to remove tumours from their inner ear, brain or spinal cord.

• #2 Neurofibromatosis Type 2 Treatment & Management: Approach Considerations, Radiation Treatment and Chemotherapy, Tumor Resection and Radiosurgery

  https://emedicine.medscape.com/article/1178283-treatment

  For individuals diagnosed with neurofibromatosis type 2 (NF2), medical care consists of routine examinations focusing on some of the potential complications related to CNS or spinal cord lesions. […] Annual neurologic assessment by a trained specialist is most useful in this clinical setting; the neurologist may detect subtle sensory or motor deficits even before the patient is aware of any difficulties. […] For patients with multiple medical problems associated with NF2, management by a team of specialists through a multidisciplinary clinic may provide the most comprehensive and cost-effective care over time. This is especially important with rapid advances in surgical management, including the use of such tools as stereotactic radiosurgery and auditory brainstem implants (ABIs). […] Although surgical resection of symptomatic tumors represents the most common approach to clinically significant lesions, in some rare instances, radiation and/or chemotherapy may be recommended to treat disabling ependymomas.

• #3 Neurofibromatosis Type 2 Treatment & Management: Approach Considerations, Radiation Treatment and Chemotherapy, Tumor Resection and Radiosurgery

  https://emedicine.medscape.com/article/1178283-treatment

  For individuals diagnosed with neurofibromatosis type 2 (NF2), medical care consists of routine examinations focusing on some of the potential complications related to CNS or spinal cord lesions. […] Annual neurologic assessment by a trained specialist is most useful in this clinical setting; the neurologist may detect subtle sensory or motor deficits even before the patient is aware of any difficulties. […] For patients with multiple medical problems associated with NF2, management by a team of specialists through a multidisciplinary clinic may provide the most comprehensive and cost-effective care over time. This is especially important with rapid advances in surgical management, including the use of such tools as stereotactic radiosurgery and auditory brainstem implants (ABIs). […] Although surgical resection of symptomatic tumors represents the most common approach to clinically significant lesions, in some rare instances, radiation and/or chemotherapy may be recommended to treat disabling ependymomas.

• #4

  https://www.advocatehealth.com/health-services/brain-spine-institute/brain-care-center/conditions-treatments/neurofibromatosis

  Theres no cure for NF1 or NF2, so neurofibromatosis treatment is aimed at minimizing symptoms. Medications can be prescribed for seizures, pain or other symptoms. […] The tumors associated with neurofibromatosis are usually benign but schwannomas and plexiform neurofibromas may rarely become cancerous. Some benign tumors cause symptoms if they grow big enough to put pressure on nerves, organs or other tissues. When these things happen, surgery or radiation may be used to remove the tumor and improve symptoms. […] Radiation is sometimes preferred for tumor removal because it can be more precise and avoid causing more damage to nerves. […] Any surgery or radiation for neurofibromatosis treatment needs to be weighed against the possibility that it will cause more damage to the nerves or tissues affected. […] Both children and adults with NF1 or NF2 have regular checkups to follow symptoms and screen for evolving problems such as hearing loss, more tumors or tumor growth. Blood tests, X-rays or other tests may be recommended to check any other concerns.

• #5 Neurofibromatosis Type 2 Diagnosis & Treatment – NYC | Columbia Neurosurgery in New York City

  https://www.neurosurgery.columbia.edu/patient-care/conditions/neurofibromatosis-type-2

  Several options are available to treat these tumors, including observation, surgical removal, peripheral nerve surgery, stereotactic radiosurgery, chemotherapy, and radiation therapy. […] At Columbia, our neurosurgeons use the latest techniques and most advanced technology to treat acoustic neuromas, and other tumors associated with NF2, to provide the best possible outcomes. […] Treatment depends on the size and type of tumor and also whether the tumor is causing symptoms. If the tumor is asymptomatic, close observation using imaging studies may be recommended in lieu of surgical intervention. […] For most brain tumors arising with NF2, the standard treatment is brain tumor surgery to resect the tumor. […] In surgery to resect acoustic neuromas, removal of tumorous tissue and protecting the patients hearing are simultaneous priorities. Small acoustic neuromas may be treated by surgical resection along with stereotactic radiosurgerya noninvasive technique in which a highly focused beam of radiation is used to destroy the tumor. Chemotherapy is sometimes also used.

• #6 Neurofibromatosis Type 2 Treatment & Management: Approach Considerations, Radiation Treatment and Chemotherapy, Tumor Resection and Radiosurgery

  https://emedicine.medscape.com/article/1178283-treatment

  Bevacizumab is an antivascular endothelial growth factor monoclonal antibody that shows some efficacy in decreasing the size of vestibular schwannomas. […] Lapatinib is a kinase inhibitor (epidermal growth factor (EGF)-receptor human epidermal growth factor 2 (HER2) kinase) that may also be considered for use in reducing the size of vestibular schwannomas and to improve hearing. […] Surgical resection of tumors remains the mainstay of treatment in neurofibromatosis type 2 (NF2), with recent advances in surgery permitting preservation of hearing for some affected individuals. […] For small vestibular schwannomas, surgical resection and stereotactic radiosurgery have been used and may preserve hearing and facial nerve function in selected patients. […] Larger tumors may require surgical resection despite irreversible hearing loss, especially when there is evidence of brainstem compression, facial nerve palsy, or, in extreme cases, early hydrocephalus.

• #7 Neurofibromatosis Type 2 | Dallas Ear Institute

  https://www.dallasear.com/education/skull-base-surgery/neurofibromatosis-type-ii/

  How Is Nf2 Treated? […] Unfortunately, there is no cure for NF2. Treatment of the various tumors that occur is recommended based the characteristics of each tumor. Some tumors may need no treatment. Others may require radiation. Some may need to be removed surgically. These decisions are complex and are best made by a team of doctors each with different areas of expertise. […] Treatment of vestibular schwannomas is required so that they do not become so large that they compress the brainstem where centers for basic functions of life occur. The brainstem plays an important role in regulation of breathing, heart rate, blood pressure and wakefulness. […] Hearing status is very important when deciding how to treat vestibular schwannomas. This is because hearing in both ears will likely be threatened over the patient’s life. If hearing is not yet affected, treatment risks worsening hearing. If hearing loss is already profound, treatment has much less risk.

• #8 Neurofibromatosis Type 2 | Condition | UT Southwestern Medical Center

  https://utswmed.org/conditions-treatments/neurofibromatosis-type-2/

  UT Southwestern Medical Centers Neurofibromatosis Type 2 (NF2) program brings together multiple specialists to provide comprehensive care for those living with this rare and complicated genetic disorder. […] While there is no cure for NF2, our multidisciplinary team can help improve a patients quality of life by lessening the debilitating consequences of the disease. Some of the treatments we provide are microsurgery, radiosurgery, chemotherapy, and hearing rehabilitation, including cochlear and auditory brainstem implants. […] In some cases, microsurgery is required for growing tumors when deafness occurs or the tumors become large enough to cause symptoms. Our neurotologists and neurosurgeons remove the intracranial tumors and offer three microsurgical approaches for the management of the vestibular schwannomas: translabyrinthine, middle fossa, and retrosigmoid craniotomies. These approaches may provide hearing preservation in some cases.

• #9 Neurofibromatosis Type 2 Treatment & Management: Approach Considerations, Radiation Treatment and Chemotherapy, Tumor Resection and Radiosurgery

  https://emedicine.medscape.com/article/1178283-treatment

  Bevacizumab is an antivascular endothelial growth factor monoclonal antibody that shows some efficacy in decreasing the size of vestibular schwannomas. […] Lapatinib is a kinase inhibitor (epidermal growth factor (EGF)-receptor human epidermal growth factor 2 (HER2) kinase) that may also be considered for use in reducing the size of vestibular schwannomas and to improve hearing. […] Surgical resection of tumors remains the mainstay of treatment in neurofibromatosis type 2 (NF2), with recent advances in surgery permitting preservation of hearing for some affected individuals. […] For small vestibular schwannomas, surgical resection and stereotactic radiosurgery have been used and may preserve hearing and facial nerve function in selected patients. […] Larger tumors may require surgical resection despite irreversible hearing loss, especially when there is evidence of brainstem compression, facial nerve palsy, or, in extreme cases, early hydrocephalus.

• #10 Neurofibromatosis Type 2: Practice Essentials, Background, Etiology

  https://emedicine.medscape.com/article/1178283-overview

  Larger tumors may require surgical resection despite irreversible hearing loss. […] A debulking procedure may result in preservation of hearing or, at the minimum, prolongation of auditory decompensation. […] Nonvestibular cranial nerve schwannomas may be treated effectively with a combination of microsurgery and radiosurgery. […] Auditory brainstem implants (ABIs) have been used successfully in some patients with hearing loss secondary to vestibular schwannomas. […] ABIs often improve the patient’s ability to appreciate environmental sounds and facilitate communication.

• #11 Neurofibromatosis Type 2 Diagnosis & Treatment – NYC | Columbia Neurosurgery in New York City

  https://www.neurosurgery.columbia.edu/patient-care/conditions/neurofibromatosis-type-2

  For other brain tumors that may arise with NF2, brain tumor surgery, chemotherapy and/or radiation therapy may be used. […] The benign peripheral nerve tumors associated with NF2 can be treated by surgical removal. Our experienced neurosurgeons meticulously dissect the tumor away from the fragile nerve, leaving the nerve intact and protecting its function. […] Treatment options for Neurofibromatosis Type 2 include Brain Tumor Surgery, Computer Assisted Surgery (CAS), Craniotomy, Gamma Knife Radiosurgery, and Microsurgery.

• #12 Neurofibromatosis – AANS

  https://www.aans.org/patients/conditions-treatments/neurofibromatosis/

  Patients with NF2 should have similar routine examinations and care. Known growths are often imaged with periodic surveillance scans with treatment reserved for enlarging or symptomatic growths. […] There is no known treatment or cure for neurofibromatosis or schwannomatosis. Medication can be prescribed to help with pain. In some cases, growths may be removed surgically or reduced with radiation therapy. Although surgery in these areas can cause further injury to nerves and additional neurological problems, it is usually well tolerated. The benefits of surgery should always be weighed against its risks. Likewise, in situations where radiation treatment is an option, the risks and benefits must be carefully considered.

• #13 Neurofibromatosis Type 2 Diagnosis & Treatment – NYC | Columbia Neurosurgery in New York City

  https://www.neurosurgery.columbia.edu/patient-care/conditions/neurofibromatosis-type-2

  Several options are available to treat these tumors, including observation, surgical removal, peripheral nerve surgery, stereotactic radiosurgery, chemotherapy, and radiation therapy. […] At Columbia, our neurosurgeons use the latest techniques and most advanced technology to treat acoustic neuromas, and other tumors associated with NF2, to provide the best possible outcomes. […] Treatment depends on the size and type of tumor and also whether the tumor is causing symptoms. If the tumor is asymptomatic, close observation using imaging studies may be recommended in lieu of surgical intervention. […] For most brain tumors arising with NF2, the standard treatment is brain tumor surgery to resect the tumor. […] In surgery to resect acoustic neuromas, removal of tumorous tissue and protecting the patients hearing are simultaneous priorities. Small acoustic neuromas may be treated by surgical resection along with stereotactic radiosurgerya noninvasive technique in which a highly focused beam of radiation is used to destroy the tumor. Chemotherapy is sometimes also used.

• #14 Neurofibromatosis Type 2 Diagnosis & Treatment – NYC | Columbia Neurosurgery in New York City

  https://www.neurosurgery.columbia.edu/patient-care/conditions/neurofibromatosis-type-2

  Several options are available to treat these tumors, including observation, surgical removal, peripheral nerve surgery, stereotactic radiosurgery, chemotherapy, and radiation therapy. […] At Columbia, our neurosurgeons use the latest techniques and most advanced technology to treat acoustic neuromas, and other tumors associated with NF2, to provide the best possible outcomes. […] Treatment depends on the size and type of tumor and also whether the tumor is causing symptoms. If the tumor is asymptomatic, close observation using imaging studies may be recommended in lieu of surgical intervention. […] For most brain tumors arising with NF2, the standard treatment is brain tumor surgery to resect the tumor. […] In surgery to resect acoustic neuromas, removal of tumorous tissue and protecting the patients hearing are simultaneous priorities. Small acoustic neuromas may be treated by surgical resection along with stereotactic radiosurgerya noninvasive technique in which a highly focused beam of radiation is used to destroy the tumor. Chemotherapy is sometimes also used.

• #15 Neurofibromatosis Type 2 Clinic – Overview – Mayo Clinic

  https://www.mayoclinic.org/departments-centers/neurofibromatosis-type-2-clinic/overview/ovc-20481871

  Neurofibromatosis type 2 (NF2) is an uncommon genetic condition that results in the development of multiple brain tumors and spinal tumors. […] At the Neurofibromatosis Type 2 Clinic at Mayo Clinic’s campus in Rochester, Minnesota, you will receive comprehensive care for the diagnosis and treatment of NF2. […] The Neurofibromatosis Type 2 Clinic provides efficient, innovative care that addresses all aspects of the condition. […] At the Neurofibromatosis Type 2 Clinic, doctors use the latest technology and techniques to treat the tumors that develop and related symptoms. Your treatment plan might involve one or more of the following approaches. […] Surgery. The type of surgery depends on the size, type, location and depth of the tumor. […] Focused radiation therapy. Radiation therapy uses high-intensity beams of energy to treat tumors, including schwannomas and meningiomas.

• #16 Neurofibromatosis: Types and Treatment | Doctor

  https://patient.info/doctor/neurofibromatosis-pro

  Neurofibromatosis type 2 requires assessment of their hearing. Ophthalmology evaluation, MRI, audiology, and brainstem evoked potentials are important in managing these patients. […] Surgery is still the first-line treatment for symptomatic tumours, but there is a 44% recurrence rate. Radiation can be used, but there is increased the risk of malignant transformation. […] Bevacizumab, a VEGF inhibitor, is a monoclonal antibody and can be used to treat neurofibromatosis type 2 patients medically. It decreased tumour size in 53% of cases and improved hearing in 57%. […] Patients with suspected neurofibromatosis type 2 should have an MRI of the head and spine done. Getting thin cuts through the internal auditory canals is important. Treatment is done if the tumour is compressing on the brainstem or preventing hearing loss.

• #17 The genetic landscape and possible therapeutics of neurofibromatosis type 2 | Cancer Cell International | Full Text

  https://cancerci.biomedcentral.com/articles/10.1186/s12935-023-02940-8

  The advent of molecular biology to scheme the pathogenesis of NF2 can guide to address of new therapeutic potentials for the effective prevention and treatment of NF2-related tumors. […] The multifocal nature of NF2-related tumors, their proximity to the vital structures (e.g., brain stem and internal carotid artery), or neural involvement (e.g., facial and auditory nerves) can limit surgical interventions. […] In patients with NF2, radiotherapy can increase the risk of developing additional benign tumors in the irradiated field and malignant transformation of existing benign tumors. […] Therefore, it is only applied in inoperable cases. […] The treatment of NF2 must start with one critical question: how much do the treatment benefits outweigh its risks? […] The available systemic therapies are limited to a handful of choices (e.g., VEGF or mTOR inhibitors) with limited efficacy.

• #18 Neurofibromatosis type 2 (NF2)

  https://www.nhs.uk/conditions/neurofibromatosis-type-2/treatment/

  Theres currently no cure for neurofibromatosis type 2 (NF2), but there are treatments that can help manage your symptoms. […] Depending on your symptoms, treatments for NF2 include: hearing aids or cochlear implants (a cochlear implant is a small device that stimulates the nerve that sends sound signals to your brain), medicine that can stop tumours growing and sometimes improve hearing, surgery to remove tumours that are at risk of causing problems, using a beam of radiation to treat small tumours or pieces of a larger tumour that are left after surgery (radiotherapy). […] Most people with NF2 will need surgery at some point during their life to remove tumours from their inner ear, brain or spinal cord.

• #19 Treatment of Patients With Advanced Neurofibromatosis Type 2 With Novel Molecularly Targeted Therapies: From Bench to Bedside

  https://pmc.ncbi.nlm.nih.gov/articles/PMC3295556/

  Minimal treatment options exist for advanced, inoperable neurofibromatosis type 2 (NF2), which is a rare tumor-prone disorder. […] Few options are available to these patients outside of surgery, which is the mainstay of treatment for NF2-associated lesions, and, in some instances, radiation therapy. […] Recently, the antivascular endothelial growth factor (VEGF) antibody bevacizumab and erlotinib exhibited promising activity in pilot trials. […] Other than these two agents, no medical options are available for patients with NF2 with surgically unresectable disease. […] Two patients were treated with a RAt sarcoma (RAS) inhibitor (salirasib), and both patients achieved stable disease (SD) for 10 and more than 52 months. […] The patient who achieved SD for more than 4.5 years while treated with the RAS inhibitor had progressive disease in his course before salirasib, which resulted in spinal cord compression with urinary incontinence and lower extremity problems that required surgery.

• #20 Neurofibromatosis Type 2 Treatment & Management: Approach Considerations, Radiation Treatment and Chemotherapy, Tumor Resection and Radiosurgery

  https://emedicine.medscape.com/article/1178283-treatment

  Bevacizumab is an antivascular endothelial growth factor monoclonal antibody that shows some efficacy in decreasing the size of vestibular schwannomas. […] Lapatinib is a kinase inhibitor (epidermal growth factor (EGF)-receptor human epidermal growth factor 2 (HER2) kinase) that may also be considered for use in reducing the size of vestibular schwannomas and to improve hearing. […] Surgical resection of tumors remains the mainstay of treatment in neurofibromatosis type 2 (NF2), with recent advances in surgery permitting preservation of hearing for some affected individuals. […] For small vestibular schwannomas, surgical resection and stereotactic radiosurgery have been used and may preserve hearing and facial nerve function in selected patients. […] Larger tumors may require surgical resection despite irreversible hearing loss, especially when there is evidence of brainstem compression, facial nerve palsy, or, in extreme cases, early hydrocephalus.

• #21 Treatment of Patients With Advanced Neurofibromatosis Type 2 With Novel Molecularly Targeted Therapies: From Bench to Bedside

  https://pmc.ncbi.nlm.nih.gov/articles/PMC3295556/

  An updated list of candidate drugs for treating NF2 was also recommended. […] Among these agents, only bevacizumab had been used to treat patients with NF2 who were considered poor candidates for surgery and radiation therapy. […] After bevacizumab treatment, tumors diminished in size in nine of 10 patients, and four of six patients had an imaging response, which was maintained for 11 to 16 months of follow-up. […] The median best response to treatment was a volumetric reduction of tumor of 26%. […] Adverse events were of grade 1 or 2 only, and thus, the drug was well-tolerated. […] In summary, novel targeted agents, alone or in combination with other treatments, are reasonable options for patients with NF2 and may allow rapid proof-of-principle translation of rational therapies.

• #22 83 Medical Therapy for Neurofibromatosis Type 2 Focusing on Vestibular Schwannoma | Neupsy Key

  https://neupsykey.com/83-medical-therapy-for-neurofibromatosis-type-2-focusing-on-vestibular-schwannoma/

  A recent multicenter, phase 2 clinical trial (NCT01207687) of 14 patients with NF2 found a confirmed hearing response in 36% of patients and radiographic response in 43% of patients, thereby confirming the efficacy and safety of bevacizumab in this subset of people with NF2 and progressive, symptomatic VSs. […] It is hypothesized that bevacizumab’s effects on reducing tumor volume is secondary to decreasing vascular permeability and decreasing outgrowth of new vessels, as opposed to having a direct antiproliferative effect. […] Unfortunately, tumor regrowth has been noted upon discontinuation of the drug, which may occur shortly after 6 months or longer following discontinuation of the drug; as a result, prolonged treatment may be necessary in order to sustain clinical benefit. […] Another ongoing clinical trial (NCT01767792) studying bevacizumab for children and young adults with hearing loss resulting from an NF2-associated VS will further investigate and expand upon the findings from this study.

• #23 The genetic landscape and possible therapeutics of neurofibromatosis type 2 | Cancer Cell International | Full Text

  https://cancerci.biomedcentral.com/articles/10.1186/s12935-023-02940-8

  Exploring its genetic and epigenetic backgrounds and the involved signaling pathways can help to find better treatments. […] Bevacizumab has shown promise in the treatment of progressive VS in NF2 patients. […] A meta-analysis of 161 patients with NF2-related VS (total of 196 VS tumors) reported that bevacizumab resulted in partial regression, stable disease, and progression in 41%, 47%, and 7%, respectively. […] The mainstay treatment of progressive meningiomas threatening functional loss is surgery. […] Bevacizumab is indicated to repress tumor growth in recurrent WHO grades II/III meningiomas. […] Emerging evidence suggests that bevacizumab can improve the symptoms of NF2-associated ependymomas. […] Surgical interventions can also be applied to improve hearing impairments due to NF2 tumors. […] The significant impacts of NF2 on the patients quality of life and the severity of symptoms urge the need to explore effective therapies.

• #24 Bevacizumab (Avastin) in NF2 (NF2-related schwannomatosis) | CUH

  https://www.cuh.nhs.uk/patient-information/bevacizumab-avastin-in-neurofibromatosis-type-ii/

  Due to the results that have been shown in the USA, since 2010 Bevacizumab has been available in the UK for treatment of rapidly growing schwannomas and symptomatic cystic ependymomas in NF2 via specialist NF2 multi-disciplinary clinic providing strict clinical criteria are met. […] The aim of receiving Bevacizumab in NF2 is to try to stop the growth of some NF2 related tumours. In some cases, patients have also found that their hearing improves. If the Bevacizumab stops the tumour(s) from growing, this may delay your need to have surgery. […] Bevacizumab is given in the Oncology Unit for adults, C9 for TYA (teenage and young adult) patients or PDU for children at Addenbrooke’s hospital in Cambridge. […] For the first six months of treatment, Avastin is given every two or three weeks and is then generally reduced to every four weeks. […] If you continue on the treatment with minimal side effects and your tumours are stable, after two years the scanning frequency may be reduced to annually and you may be offered an elective break in treatment. This will be discussed with you at the appropriate time.

• #25 Treatment of Patients With Advanced Neurofibromatosis Type 2 With Novel Molecularly Targeted Therapies: From Bench to Bedside

  https://pmc.ncbi.nlm.nih.gov/articles/PMC3295556/

  An updated list of candidate drugs for treating NF2 was also recommended. […] Among these agents, only bevacizumab had been used to treat patients with NF2 who were considered poor candidates for surgery and radiation therapy. […] After bevacizumab treatment, tumors diminished in size in nine of 10 patients, and four of six patients had an imaging response, which was maintained for 11 to 16 months of follow-up. […] The median best response to treatment was a volumetric reduction of tumor of 26%. […] Adverse events were of grade 1 or 2 only, and thus, the drug was well-tolerated. […] In summary, novel targeted agents, alone or in combination with other treatments, are reasonable options for patients with NF2 and may allow rapid proof-of-principle translation of rational therapies.

• #26 83 Medical Therapy for Neurofibromatosis Type 2 Focusing on Vestibular Schwannoma | Neupsy Key

  https://neupsykey.com/83-medical-therapy-for-neurofibromatosis-type-2-focusing-on-vestibular-schwannoma/

  A recent multicenter, phase 2 clinical trial (NCT01207687) of 14 patients with NF2 found a confirmed hearing response in 36% of patients and radiographic response in 43% of patients, thereby confirming the efficacy and safety of bevacizumab in this subset of people with NF2 and progressive, symptomatic VSs. […] It is hypothesized that bevacizumab’s effects on reducing tumor volume is secondary to decreasing vascular permeability and decreasing outgrowth of new vessels, as opposed to having a direct antiproliferative effect. […] Unfortunately, tumor regrowth has been noted upon discontinuation of the drug, which may occur shortly after 6 months or longer following discontinuation of the drug; as a result, prolonged treatment may be necessary in order to sustain clinical benefit. […] Another ongoing clinical trial (NCT01767792) studying bevacizumab for children and young adults with hearing loss resulting from an NF2-associated VS will further investigate and expand upon the findings from this study.

• #27 Neurofibromatosis Type 2 Treatment & Management: Approach Considerations, Radiation Treatment and Chemotherapy, Tumor Resection and Radiosurgery

  https://emedicine.medscape.com/article/1178283-treatment

  Bevacizumab is an antivascular endothelial growth factor monoclonal antibody that shows some efficacy in decreasing the size of vestibular schwannomas. […] Lapatinib is a kinase inhibitor (epidermal growth factor (EGF)-receptor human epidermal growth factor 2 (HER2) kinase) that may also be considered for use in reducing the size of vestibular schwannomas and to improve hearing. […] Surgical resection of tumors remains the mainstay of treatment in neurofibromatosis type 2 (NF2), with recent advances in surgery permitting preservation of hearing for some affected individuals. […] For small vestibular schwannomas, surgical resection and stereotactic radiosurgery have been used and may preserve hearing and facial nerve function in selected patients. […] Larger tumors may require surgical resection despite irreversible hearing loss, especially when there is evidence of brainstem compression, facial nerve palsy, or, in extreme cases, early hydrocephalus.

• #28 Treatment of Patients With Advanced Neurofibromatosis Type 2 With Novel Molecularly Targeted Therapies: From Bench to Bedside

  https://pmc.ncbi.nlm.nih.gov/articles/PMC3295556/

  Minimal treatment options exist for advanced, inoperable neurofibromatosis type 2 (NF2), which is a rare tumor-prone disorder. […] Few options are available to these patients outside of surgery, which is the mainstay of treatment for NF2-associated lesions, and, in some instances, radiation therapy. […] Recently, the antivascular endothelial growth factor (VEGF) antibody bevacizumab and erlotinib exhibited promising activity in pilot trials. […] Other than these two agents, no medical options are available for patients with NF2 with surgically unresectable disease. […] Two patients were treated with a RAt sarcoma (RAS) inhibitor (salirasib), and both patients achieved stable disease (SD) for 10 and more than 52 months. […] The patient who achieved SD for more than 4.5 years while treated with the RAS inhibitor had progressive disease in his course before salirasib, which resulted in spinal cord compression with urinary incontinence and lower extremity problems that required surgery.

• #29 Treatment of Patients With Advanced Neurofibromatosis Type 2 With Novel Molecularly Targeted Therapies: From Bench to Bedside

  https://pmc.ncbi.nlm.nih.gov/articles/PMC3295556/

  Interestingly, after receiving the RAS inhibitor, the patient had no additional disease progression. […] One patient had SD after treatment with a mitogen-activated protein kinase 1 inhibitor (mitogen-activated protein/extracellular signal-regulated kinase or MAP/ERK kinase1 inhibitor) for 7 months. […] The patient subsequently had ongoing SD in response to bevacizumab, which is a VEGF antibody, for more than 22 months. […] Two other patients with NF2 treated with bevacizumab and an mTOR inhibitor combination had SD for more than 4 and 9 months. […] The patient who had SD by RECIST for more than 9 months has, thus far, had a 33% decrease in tumor size by volumetric analysis. […] Adverse-effect profiles of the patients are outlined in Table 2. […] Our results showed that patients with NF2 who were referred to a clinical trials center for targeted therapy treatment demonstrated acceptable safety profiles and preliminary evidence of activity, and targeted therapy is a pragmatic option in this rare-disease setting.

• #30 Treatment of Patients With Advanced Neurofibromatosis Type 2 With Novel Molecularly Targeted Therapies: From Bench to Bedside

  https://pmc.ncbi.nlm.nih.gov/articles/PMC3295556/

  Interestingly, after receiving the RAS inhibitor, the patient had no additional disease progression. […] One patient had SD after treatment with a mitogen-activated protein kinase 1 inhibitor (mitogen-activated protein/extracellular signal-regulated kinase or MAP/ERK kinase1 inhibitor) for 7 months. […] The patient subsequently had ongoing SD in response to bevacizumab, which is a VEGF antibody, for more than 22 months. […] Two other patients with NF2 treated with bevacizumab and an mTOR inhibitor combination had SD for more than 4 and 9 months. […] The patient who had SD by RECIST for more than 9 months has, thus far, had a 33% decrease in tumor size by volumetric analysis. […] Adverse-effect profiles of the patients are outlined in Table 2. […] Our results showed that patients with NF2 who were referred to a clinical trials center for targeted therapy treatment demonstrated acceptable safety profiles and preliminary evidence of activity, and targeted therapy is a pragmatic option in this rare-disease setting.

• #31 Repurposed Drug Shrinks NF2-related Schwannomatosis Tumors – InventUMPage 1arrow–buttonPage 1arrow–buttonPage 1arrow–buttonPage 1arrow–buttonPage 1arrow–button

  https://news.med.miami.edu/repurposed-drug-shrinks-nf2-related-schwannomatosis-tumors/

  Researchers have evidence that the approved lung cancer drug, brigatinib, may shrink tumors associated with neurofibromatosis type 2 (NF2)-related schwannomatosis. […] The study confirmed 10% of growing tumors and 23% of all tumors shrank and one-third of patients experienced improvement in hearing and pain. […] There are no approved treatments for NF2-related schwannomatosis. Doctors and surgeons manage progressive schwannomas meningiomas, and ependymomas associated with the syndrome with surgery, radiation or off-label chemotherapy. […] The research team found that in response to the small molecule brigatinib, 10% of growing tumors and 23% of all tumors in those studied shrank. Up to one-third of patients experienced objective improvement in hearing and reductions in pain. […] Brigatinib is a well-tolerated oral medication.

• #32 Treatment with Brigatinib Results in Radiographic Responses

  https://www.esmo.org/oncology-news/treatment-with-brigatinib-results-in-radiographic-responses-and-clinical-benefit-in-a-heavily-pretreated-cohort-of-patients-with-nf2-related-schwannomatosis

  In a prospective phase II, adaptive platformbasket INTUITT-NF2 study, brigatinib showed broad antitumour activity in patients with NF2-related schwannomatosis who had progressive tumours. The study investigators documented the greatest activity for meningiomas and nonvestibular schwannomas, with responses in 25% and 20% of tumours. Treatment with brigatinib resulted in a decrease in the growth of all tumour types, most prominently for meningiomas. […] Treatment with brigatinib was safe and associated with improvement in hearing, and a decrease in pain. […] The authors commented that an oral agent that treats multiple types of NF2-associated tumours and has an acceptable side-effect profile for long-term use would reduce the need for high-risk interventions and avert subsequent complications in patients with NF2-related schwannomatosis. In this prospective study of brigatinib in patients with NF2-related schwannomatosis and progressive tumours, treatment with brigatinib was safe and associated with shrinkage of multiple tumour types, improvement in hearing, and a decrease in pain.

• #33

  https://journals.lww.com/anti-cancerdrugs/fulltext/2023/11000/treatment_of_neurofibromatosis_type_ii_with.1.aspx

  Patients with neurofibromatosis type II (NF2) usually require surgical treatment, but the probability of tumor recurrence remains high after surgical resection. Moreover, because most of NF2 lesions involve the facial nerve, the risk of facial nerve injury during the surgery is high. Stereotactic radiotherapy can be used to treat some cases of NF2. However, it is not recommended for treatment of multiple or large tumors, and surgical resection may be more difficult after radiotherapy. Few systemic treatments are available. At present, bevacizumab is considered the first-line drug treatment for fast-growing NF2. However, bevacizumab requires long-term administration, and tumor growth will resume after drug withdrawal. […] Here, we present a case of NF2 that developed exacerbations after multiple treatments with gamma knife and surgery, and achieved good results after later treatment with anlotinib. Accordingly, we propose that anlotinib may be a valuable treatment option for NF2.

• #34 Treatment with Brigatinib Results in Radiographic Responses

  https://www.esmo.org/oncology-news/treatment-with-brigatinib-results-in-radiographic-responses-and-clinical-benefit-in-a-heavily-pretreated-cohort-of-patients-with-nf2-related-schwannomatosis

  In a prospective phase II, adaptive platformbasket INTUITT-NF2 study, brigatinib showed broad antitumour activity in patients with NF2-related schwannomatosis who had progressive tumours. The study investigators documented the greatest activity for meningiomas and nonvestibular schwannomas, with responses in 25% and 20% of tumours. Treatment with brigatinib resulted in a decrease in the growth of all tumour types, most prominently for meningiomas. […] Treatment with brigatinib was safe and associated with improvement in hearing, and a decrease in pain. […] The authors commented that an oral agent that treats multiple types of NF2-associated tumours and has an acceptable side-effect profile for long-term use would reduce the need for high-risk interventions and avert subsequent complications in patients with NF2-related schwannomatosis. In this prospective study of brigatinib in patients with NF2-related schwannomatosis and progressive tumours, treatment with brigatinib was safe and associated with shrinkage of multiple tumour types, improvement in hearing, and a decrease in pain.

• #35 Neurofibromatosis Type 2 Clinic – Overview – Mayo Clinic

  https://www.mayoclinic.org/departments-centers/neurofibromatosis-type-2-clinic/overview/ovc-20481871

  Chemotherapy. Chemotherapy uses drugs to stop tumor growth and, in some cases, to improve certain signs and symptoms of NF2, such as hearing loss. […] Reconstructive surgery. Depending on the size and location of the tumors, some people may need reconstructive surgery to restore facial nerve function and eye closure. […] Rehabilitation. Rehabilitation specialists in speech therapy, swallowing therapy, balance therapy, dietetics, physical therapy and occupational therapy help with relevant rehabilitation. […] At the Neurofibromatosis Type 2 Clinic, experts work together as a multispecialty team to provide exactly the care you need.

• #36 Neurofibromatosis Type 2 | Boston Children’s Hospital

  https://www.childrenshospital.org/conditions/neurofibromatosis-type-2

  Hearing aids or an FM system may be recommended for use if there is documented decrease in hearing. […] In most cases, the vestibular schwannomas that grow on the auditory nerve will need to be removed surgically. […] Individuals who experience hearing loss or impairment have the following non-surgical treatment options: lip reading, sign language, hearing aids, cochlear implants. […] After an adolescent or young adult has been diagnosed with NF2, the doctors in our Neurofibromatosis Program will see her at least once per year and arrange more frequent follow-up visits if necessary. […] The most important things a parent can do for a child who has NF2 is make sure that he is comfortable and safe in his environment, and encourage him to participate in physical therapy and mobility training.

• #37 Treatment & Management – Neurofibromatosis Program

  https://www.uab.edu/medicine/nfprogram/learn-about-nf/nf2/treatment-management

  Radiation therapy The goal of this approach is to reduce the size of tumors in order to preserve hearing for as long as possible and lessen the severity of neurological complications. Radiation may be given in one singe treatment or in several smaller treatments over a period of time and may include use of a highly focused beam of radiation called stereotactic radiation therapy. […] Treatments for hearing loss Three types of hearing interventions the BAHA hearing aid, the cochlear implant, and the auditory brainstem implant can sometimes restore at least partial hearing for some people with NF2. The BAHA (bone-anchored hearing aid) is surgically implanted under the skin near the ear; an external speech processor transmits sound vibrations within the skull and inner ear, simulating the nerves of the ear and allowing the patient to hear. Cochlear implants might be used in NF2 patients with bilateral hearing loss. The implant consists of a device surgically implanted under the skin near the ear as well as an external earpiece. The device bypasses damaged portions of the ear, directly stimulating the hearing nerve and generating signals from the auditory nerve to the brain. The brainstem implant transmits the sound directly to the brain, and may be more applicable to patients with NF2 than the cochlear implant because it completely bypasses the site of the tumor.

• #38 Neurofibromatosis Type 2 | Boston Children’s Hospital

  https://www.childrenshospital.org/conditions/neurofibromatosis-type-2

  Hearing aids or an FM system may be recommended for use if there is documented decrease in hearing. […] In most cases, the vestibular schwannomas that grow on the auditory nerve will need to be removed surgically. […] Individuals who experience hearing loss or impairment have the following non-surgical treatment options: lip reading, sign language, hearing aids, cochlear implants. […] After an adolescent or young adult has been diagnosed with NF2, the doctors in our Neurofibromatosis Program will see her at least once per year and arrange more frequent follow-up visits if necessary. […] The most important things a parent can do for a child who has NF2 is make sure that he is comfortable and safe in his environment, and encourage him to participate in physical therapy and mobility training.

• #39 Neurofibromatosis Type 2 | Boston Children’s Hospital

  https://www.childrenshospital.org/conditions/neurofibromatosis-type-2

  Since a cure for NF2 has not yet been found, treatment focuses on managing symptoms and complications. Experts here at Children’s will customize a comprehensive treatment plan based on each patient’s unique needs. […] A person with NF2 might need one or more of the following treatment methods in order to manage symptoms: […] Surgery may be used to remove schwannomas that are pushing on the spinal cord or meningiomas (a non-cancerous tumor) that are causing seizures. […] Medication can also sometimes be useful in managing your child’s symptoms; for example, if she’s experiencing ringing in the ears (tinnitus), headaches or pain caused by tumors pressing on her nerves. […] Children with NF2 may have a tumor pushing on the cerebellum (the part of brain that controls balance). These tumors usually need to be surgically removed in order for the problem to be eliminated. However, physical therapy (PT) can compensate for problems with balance and weakness by helping to strengthen the core muscles.

• #40 Neurofibromatosis Type 2 Treatment | UVA Health

  https://uvahealth.com/services/neurocutaneous/neurofibromatosis-type-2

  Theres no cure for neurofibromatosis type 2, but our experienced team at UVA Health can help make the symptoms better. […] Our team includes all the different specialists you need neurologists, neurosurgeons, ophthalmologists (eye doctors), otolaryngologists (ear, nose, and throat doctors), and therapists. […] Well work with you to make a personalized treatment plan that meets your individual needs and gives you the greatest quality of life. […] If your tumors are small, we may recommend watchful waiting. […] If your tumors cause hearing loss or other problems, we can do surgery to remove them. […] Gamma Knife uses focused radiation on tumors without hurting the tissue nearby. […] Hearing aids, cochlear implants, and vision aids can help you communicate better and give you higher quality of life. […] We offer therapy services to help with balance, coordination, and communication. These include physical, occupational, and speech therapy. […] Our social workers can help you work through the emotional impacts of living with a chronic illness.

• #41 Neurofibromatosis Treatment Pipeline is Evolving with Potential Therapies

  https://www.delveinsight.com/blog/neurofibromatosis-treatment-outlook

  Type 2 Neurofibromatosis medication includes an off-label treatment with the vascular endothelial growth factor (VEGF) inhibitor bevacizumab that has now become an important therapeutic option. At least over the intermediate-term, it is associated with hearing improvement in 57% and tumor shrinkage in 55% of patients, although there is a risk of side effects including hypertension and proteinuria. Apart from that surgery is always the last treatment option. […] Schwannomatosis treatment involves functionally limiting schwannomas that can be surgically resected. Genetic investigation of the resected tumor material is of high prognostic relevance, in particular when the diagnosis is initially unclear. Co-analgetic drugs (amitriptyline, gabapentin, pregabalin) have been found especially useful in Neurofibromatosis pain management associated with neuropathic pain.

• #42 Neurofibromatosis Type 2 Treatment & Management: Approach Considerations, Radiation Treatment and Chemotherapy, Tumor Resection and Radiosurgery

  https://emedicine.medscape.com/article/1178283-treatment

  Annual neurologic examination looking for subtle deficits or changes in neurologic status that might suggest disease progression. […] Annual hearing screening with BAER, with referral to an audiologist for amplification, augmentation, or speech therapy recommendations. […] Annual MRI to monitor existing lesions or look for presymptomatic lesions. […] Annual ophthalmologic evaluations to monitor visual acuity.

• #43 Neurofibromatosis Type 2 Treatment & Management: Approach Considerations, Radiation Treatment and Chemotherapy, Tumor Resection and Radiosurgery

  https://emedicine.medscape.com/article/1178283-treatment

  Annual neurologic examination looking for subtle deficits or changes in neurologic status that might suggest disease progression. […] Annual hearing screening with BAER, with referral to an audiologist for amplification, augmentation, or speech therapy recommendations. […] Annual MRI to monitor existing lesions or look for presymptomatic lesions. […] Annual ophthalmologic evaluations to monitor visual acuity.

• #44 Neurofibromatosis Type 2 Treatment & Management: Approach Considerations, Radiation Treatment and Chemotherapy, Tumor Resection and Radiosurgery

  https://emedicine.medscape.com/article/1178283-treatment

  In addition, bevacizumab can be used in cases of residual tumor in patients in which complete resection is not possible. […] ABIs have been used successfully in some patients with hearing loss secondary to vestibular schwannomas. […] The neurologist and neurosurgeon work closely together in the management of central and spinal cord lesions in neurofibromatosis type 2 (NF2). […] The otolaryngologist or otologist is an important consultant in the surgical management of vestibular schwannomas, especially if ABIs are being considered. […] The audiologist serves as an essential member of the management team for individuals with acoustic nerve lesions. […] The ophthalmologist is an important team member and can assist in the diagnosis and care of the patient with NF2. […] Finally, the geneticist may provide diagnostic and genetic information to affected and at-risk individuals.

• #45 Neurofibromatosis type 2 (NF2): A clinical and molecular review | Orphanet Journal of Rare Diseases | Full Text

  https://ojrd.biomedcentral.com/articles/10.1186/1750-1172-4-16

  The progress being made in cellular research especially with regard to pathways in which the NF2 gene product interacts raises the hopes of targeted therapy. […] NF2 remains a life limiting and life spoiling condition. […] Multidisciplinary management with early diagnosis are mainstays of management. Hopefully new targeted therapies will revolutionise the outcomes in this condition.

• #46 Ending NF2 through Gene Therapy – Myriad Canada – International philanthropic network that brings together donors, philanthropists, and organizations

  https://www.myriadcanada.org/en/project/ending-nf2-through-gene-therapy/

  Current treatment options include surgery, chemo therapy and radiation. History has shown us that tumors frequently grow back following surgery. The surgery itself (often 12 hours or more) poses many risks aside from facial paralysis and other issues. There is a limit to how much radiation the brain stem can withstand and is not recommended for children as their brains are still maturing. All of these treatments are designed to try to control or slowdown tumor growth. None of them will cure the root cause of NF2. […] The goal of gene therapy is synthesize the correct copy of the defective NF2 gene in the laboratory, insert it into the body where it will knock out the defective gene and put the healthy copy in its place, thus curing the disorder. Treated people will know that from then on, their NF2 will not cause any further damage. Can you imagine what this means to a newly diagnosed child? It will be a life changer! […] Because NF2 is a mono-genetic disorder (caused by a single defective gene), it has been identified as an ideal candidate for gene therapy.

• #47

  https://link.springer.com/article/10.1007/s40487-024-00279-2

  Recently, AAV-based gene replacement therapy has displayed encouraging efficacy in a preclinical schwannoma sciatic nerve xenograft mouse model of NF2-related schwannomatosis. Thus, gene therapy holds potential as an effective and promising therapeutic strategy for NF2-related schwannomatosis in the future. […] New advances in molecular biology and pathogenic genetics study on NF2-related schwannomatosis help to establish novel gene replacement therapy to restore merlin expression, potentially providing an effective treatment for the disease. […] Currently, gene therapy approaches for NF2-related schwannomatosis primarily include suicide gene therapy, gene replacement or augmentation therapy, and gene knockdown and replacement combination approach. […] Although these gene therapy strategies have shown potential in preclinical animal model studies, they still face many specific challenges apart from the traditional challenges for gene therapy such as immunogenicity, delivery vector, manufacturing, and the long-term effects of treatments.

• #48

  https://link.springer.com/article/10.1007/s40487-024-00279-2

  Gene replacement therapy for NF2-related schwannomatosis directly supplies a functional copy of the mutated or inactivated NF2 gene to augment functional merlin protein re-expression in NF2-deficient tumor cells to treat the disease phenotype caused by the defective NF2 gene. […] This approach can not only treat existing tumors but may also be used as a preventative prophylactic gene therapy to inhibit future tumor formation by delivering AAVmerlin vectors intravenously and intrathecally with access to NF2 heterozygous cells throughout the body. Therefore, the effectiveness of gene replacement therapy could be expanded by transferring merlin via EVs or by delivering AAVmerlin vectors intravenously and intrathecally. Gene replacement therapy can also be combined with surgical resection and other currently available treatments to achieve potentially synergistic and additive therapeutic effects.

• #49 Research Sponsored – Accelerating Novel Therapy Research for Neurofibromatosis Type 2

  https://nf2biosolutions.org/gene-therapy-and-nf2/research-initiatives/

  The goal of this project is to develop a genetically modified version of the cellular product armed with genes that enhance tumor cell death and the development of anti-tumor adaptive immunity. […] NF2 BioSolutions is also supporting Dr. Gary Brenners lab to move towards a toxicology study for its Suicide gene therapy approach, prior to submitting an IND (investigational new drug) application to the FDA. […] The focus of the lab is to address this need by developing a gene therapy that specifically targets schwannomas, which are composed entirely of Schwann-lineage tumor cells. […] The team designed a gene therapy strategy that uses an adeno-associated (AAV) viral vector that expresses the pro-apoptotic gene ASC (ASC =apoptosis-associated speck-like protein containing a carboxy-terminal CARD) under the Schwann-lineage specific promoter, P0.

• #50 Research Sponsored – Accelerating Novel Therapy Research for Neurofibromatosis Type 2

  https://nf2biosolutions.org/gene-therapy-and-nf2/research-initiatives/

  NF2 BioSolutions is inviting you to participate in a study with the purpose to develop automated 3D volumetric measurements of the NF2 vestibular schwannomas from the patients MRIs. […] The advantage of his platform is its ability to silence the mutated gene and then add a healthy one. […] To introduce new copies of the NF2 gene using AAV in order to increase the amount of normal NF2 gene product (Merlin) to a level at which it can execute its tumor suppressor functions in patients. […] NF2 BioSolutions is excited to announce the funding of Dr. Gary Brenners lab at Massachusetts General Hospital for the development of immunotherapy for treating NF2. This new approach utilizes a cell-based treatment strategy, meaning that cells or cellular material are injected or implanted into a patient.

• #51 Simultaneous inhibition of PI3K and PAK in preclinical models of neurofibromatosis type 2-related schwannomatosis | Oncogene

  https://www.nature.com/articles/s41388-024-02958-w

  We have previously shown that simultaneous inhibition of complementary pathways exposes vulnerable targets due to synergistic effects. The use of high throughput viability and synergy analyses to test a large number of inhibitors was beneficial in identifying a single favorable combination that induced similar responses across a number of cell lines in multiple species. […] Pictilisib is an orally available small-molecule inhibitor for all four classes of PI3K isoforms. […] Therefore, drug combination approaches have been explored. […] In our in vivo study, we did not observe higher mice lethality in treated groups. […] A thorough understanding of the complex and intersecting molecular pathways involved could identify new targets and inhibitors to slow NF2 schwannoma disease progression. The results support the use of combinatorial treatments at potentially lower than maximally tolerated doses of monotherapies to leverage the cooperative properties of targeted inhibitors.

• #52 Neurofibromatosis Research Program, NFRP Supported Clinical Trials; Congressionally Directed Medical Research Programs

  https://cdmrp.health.mil/nfrp/clinical_trials/nf2

  Neurofibromatosis (NF) refers to a group of three genetically distinct disorders that have a spectrum of clinical manifestations. Treatments for NF currently include surgery, radiation, and chemotherapy, but several clinical trials are underway to identify more treatment options. […] The Congressionally Directed Medical Research Programs’ Neurofibromatosis Research Program (NFRP) is funding several efforts focused on finding better treatments through two main mechanisms, the Clinical Trial Award and the NF Clinical Trials Consortium. […] NF160065: Prospective, Randomized, Placebo-Controlled Phase 2 Trial of Aspirin for Vestibular Schwannomas Summary: This study is a prospective phase 2 trial with the objectives to evaluate whether the administration of aspirin can prevent growth of vestibular schwannomas, as well as determine whether biomarkers from patients blood would be able to predict a response to aspirin treatment. Status: Recruiting ClinicalTrials.gov Identifier: NCT03079999

• #53 Innovative Trial for Understanding the Impact of Targeted Therapies in Neurofibromatosis Type 2 – Related Schwannomatosis, The INTUITT-NF2 Trial – NCIFacebookFollow on XInstagramYoutubeLinkedin

  https://www.cancer.gov/about-cancer/treatment/clinical-trials/search/v?id=NCI-2020-04572

  This phase II trial investigates targeted therapies and how well they work for patients with neurofibromatosis type 2 (NF2)-related Schwannomatosis (SWN). […] The brigatinib sub-study investigates a drug called brigatinib, which acts by blocking the activity of growth pathways in tumors. Because tumors need these signals to grow, brigatinib may slow or reverse the growth of the tumors that develop in people with neurofibromatosis type 2. […] The neratinib sub-study investigates a drug called neratinib. Neratinib is a type of inhibitor that inhibits epidermal growth factor receptor (EGFR), human epidermal growth factor receptor 2 (HER2), and HER4. By stopping this signaling from working, neratinib may cause tumor cells to stop growing. […] Patients receive brigatinib orally (PO) once daily (QD) on days 1-28 of each cycle. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. […] Patients receive neratinib PO QD on days 1-28 of each cycle. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.

• #54 Neurofibromatosis II

  https://www.uchealth.com/en/conditions/neurofibromatosis-II

  Managing Neurofibromatosis II (NF2) involves a combination of treatment strategies tailored to individual patient needs. The primary goal is to alleviate symptoms and manage tumor growth effectively. […] Some drugs may be effective in controlling tumor growth while maintaining hearing. Active clinical trials are also taking place in finding novel medications. It is important for patients to consult with their care team to obtain the most up to date information. […] Surgical resection is sometimes necessary for tumors based on growth rate and hearing. […] Regular Imaging: Ongoing monitoring through MRI scans is essential to track tumor growth and detect new tumors early. […] Patients may benefit from therapies aimed at managing symptoms such as tinnitus, balance issues, and headaches. […] These treatment options aim to enhance the quality of life for NF2 patients while addressing the unique challenges posed by the condition. Regular consultations with a multidisciplinary team are crucial for optimal management.

• #55 Neurofibromatosis Type 2 Diagnosis & Treatment – NYC | Columbia Neurosurgery in New York City

  https://www.neurosurgery.columbia.edu/patient-care/conditions/neurofibromatosis-type-2

  Several options are available to treat these tumors, including observation, surgical removal, peripheral nerve surgery, stereotactic radiosurgery, chemotherapy, and radiation therapy. […] At Columbia, our neurosurgeons use the latest techniques and most advanced technology to treat acoustic neuromas, and other tumors associated with NF2, to provide the best possible outcomes. […] Treatment depends on the size and type of tumor and also whether the tumor is causing symptoms. If the tumor is asymptomatic, close observation using imaging studies may be recommended in lieu of surgical intervention. […] For most brain tumors arising with NF2, the standard treatment is brain tumor surgery to resect the tumor. […] In surgery to resect acoustic neuromas, removal of tumorous tissue and protecting the patients hearing are simultaneous priorities. Small acoustic neuromas may be treated by surgical resection along with stereotactic radiosurgerya noninvasive technique in which a highly focused beam of radiation is used to destroy the tumor. Chemotherapy is sometimes also used.

• #56 Neurofibromatosis type 2 (NF2): A clinical and molecular review | Orphanet Journal of Rare Diseases | Full Text

  https://ojrd.biomedcentral.com/articles/10.1186/1750-1172-4-16

  Neurofibromatosis type 2 is a dominantly inherited tumour predisposition syndrome caused by mutations in the NF2 gene on chromosome 22. […] Diagnosis is based on clinical and neuroimaging studies. Presymptomatic genetic testing is an integral part of the management of NF2 families. […] Surgery remains the focus of current management although watchful waiting with careful surveillance and occasionally radiation treatment have a role. […] The cornerstone of modern NF2 management is conservation of function, and the maintenance of „quality of life”. […] It is important to balance the use of microsurgery and radiation treatment, which has a role in patients who are poor surgical candidates, or who refuse or wish to avoid surgery because of its associated risks. […] The NF2 protein appears to impact on multiple intracellular signalling pathways.

• #57 Neurofibromatosis type 2 (NF2): A clinical and molecular review | Orphanet Journal of Rare Diseases | Full Text

  https://ojrd.biomedcentral.com/articles/10.1186/1750-1172-4-16

  Neurofibromatosis type 2 is a dominantly inherited tumour predisposition syndrome caused by mutations in the NF2 gene on chromosome 22. […] Diagnosis is based on clinical and neuroimaging studies. Presymptomatic genetic testing is an integral part of the management of NF2 families. […] Surgery remains the focus of current management although watchful waiting with careful surveillance and occasionally radiation treatment have a role. […] The cornerstone of modern NF2 management is conservation of function, and the maintenance of „quality of life”. […] It is important to balance the use of microsurgery and radiation treatment, which has a role in patients who are poor surgical candidates, or who refuse or wish to avoid surgery because of its associated risks. […] The NF2 protein appears to impact on multiple intracellular signalling pathways.

• #58 Neurofibromatosis type 2 (NF2): A clinical and molecular review | Orphanet Journal of Rare Diseases | Full Text

  https://ojrd.biomedcentral.com/articles/10.1186/1750-1172-4-16

  The progress being made in cellular research especially with regard to pathways in which the NF2 gene product interacts raises the hopes of targeted therapy. […] NF2 remains a life limiting and life spoiling condition. […] Multidisciplinary management with early diagnosis are mainstays of management. Hopefully new targeted therapies will revolutionise the outcomes in this condition.

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