Comprehensive, easy-to-understand information about this condition
How we create this content →The documentation surrounding isolated bone marrow mastocytosis is limited due to its rarity, affecting fewer than 20,000 individuals in the U.S. This rarity restricts the ability to conduct large-scale clinical studies and gather extensive patient data. Additionally, the condition's clinical features may overlap with other mast cell disorders, complicating characterization. Ongoing research aims to improve understanding and management of this condition.
To navigate your care for isolated bone marrow mastocytosis, consider consulting a hematologist with expertise in mast cell disorders. They can provide specialized insights into your condition and treatment options. Additionally, you may want to explore clinical trial participation for access to new therapies. For resources, visit the National Organization for Rare Disorders (NORD) at rarediseases.org, which offers valuable information and support for rare disease patients. Although no patient organizations are currently identified, staying connected with healthcare professionals and research updates is crucial.
There are several FDA-approved treatments for isolated bone marrow mastocytosis, including avapritinib, Cromolyn sodium, and midostaurin. Additionally, there are multiple orphan drugs designated for development, such as bezuclastinib and various targeted therapies. Currently, there are 4 active clinical trials exploring these treatments. For more information, you can search for ongoing trials at ClinicalTrials.gov using the following link: https://clinicaltrials.gov/search?cond=isolated%20bone%20marrow%20mastocytosis.
Actionable guidance for navigating care for isolated bone marrow mastocytosis
To navigate your care for isolated bone marrow mastocytosis, consider consulting a hematologist with expertise in mast cell disorders. They can provide specialized insights into your condition and treatment options. Additionally, you may want to explore clinical trial participation for access to new therapies. For resources, visit the National Organization for Rare Disorders (NORD) at rarediseases.org, which offers valuable information and support for rare disease patients. Although no patient organizations are currently identified, staying connected with healthcare professionals and research updates is crucial.
Consider asking your healthcare providers these condition-specific questions
Helpful links for rare disease information and support
The documentation surrounding isolated bone marrow mastocytosis is limited due to its rarity, affecting fewer than 20,000 individuals in the U.S. This rarity restricts the ability to conduct large-scale clinical studies and gather extensive patient data. Additionally, the condition's clinical features may overlap with other mast cell disorders, complicating characterization. Ongoing research aims to improve understanding and management of this condition.
To navigate your care for isolated bone marrow mastocytosis, consider consulting a hematologist with expertise in mast cell disorders. They can provide specialized insights into your condition and treatment options. Additionally, you may want to explore clinical trial participation for access to new therapies. For resources, visit the National Organization for Rare Disorders (NORD) at rarediseases.org, which offers valuable information and support for rare disease patients. Although no patient organizations are currently identified, staying connected with healthcare professionals and research updates is crucial.
There are several FDA-approved treatments for isolated bone marrow mastocytosis, including avapritinib, Cromolyn sodium, and midostaurin. Additionally, there are multiple orphan drugs designated for development, such as bezuclastinib and various targeted therapies. Currently, there are 4 active clinical trials exploring these treatments. For more information, you can search for ongoing trials at ClinicalTrials.gov using the following link: https://clinicaltrials.gov/search?cond=isolated%20bone%20marrow%20mastocytosis.
Actionable guidance for navigating care for isolated bone marrow mastocytosis
To navigate your care for isolated bone marrow mastocytosis, consider consulting a hematologist with expertise in mast cell disorders. They can provide specialized insights into your condition and treatment options. Additionally, you may want to explore clinical trial participation for access to new therapies. For resources, visit the National Organization for Rare Disorders (NORD) at rarediseases.org, which offers valuable information and support for rare disease patients. Although no patient organizations are currently identified, staying connected with healthcare professionals and research updates is crucial.
Consider asking your healthcare providers these condition-specific questions
Helpful links for rare disease information and support
The documentation surrounding isolated bone marrow mastocytosis is limited due to its rarity, affecting fewer than 20,000 individuals in the U.S. This rarity restricts the ability to conduct large-scale clinical studies and gather extensive patient data. Additionally, the condition's clinical features may overlap with other mast cell disorders, complicating characterization. Ongoing research aims to improve understanding and management of this condition.
To navigate your care for isolated bone marrow mastocytosis, consider consulting a hematologist with expertise in mast cell disorders. They can provide specialized insights into your condition and treatment options. Additionally, you may want to explore clinical trial participation for access to new therapies. For resources, visit the National Organization for Rare Disorders (NORD) at rarediseases.org, which offers valuable information and support for rare disease patients. Although no patient organizations are currently identified, staying connected with healthcare professionals and research updates is crucial.
There are several FDA-approved treatments for isolated bone marrow mastocytosis, including avapritinib, Cromolyn sodium, and midostaurin. Additionally, there are multiple orphan drugs designated for development, such as bezuclastinib and various targeted therapies. Currently, there are 4 active clinical trials exploring these treatments. For more information, you can search for ongoing trials at ClinicalTrials.gov using the following link: https://clinicaltrials.gov/search?cond=isolated%20bone%20marrow%20mastocytosis.
Actionable guidance for navigating care for isolated bone marrow mastocytosis
To navigate your care for isolated bone marrow mastocytosis, consider consulting a hematologist with expertise in mast cell disorders. They can provide specialized insights into your condition and treatment options. Additionally, you may want to explore clinical trial participation for access to new therapies. For resources, visit the National Organization for Rare Disorders (NORD) at rarediseases.org, which offers valuable information and support for rare disease patients. Although no patient organizations are currently identified, staying connected with healthcare professionals and research updates is crucial.
Consider asking your healthcare providers these condition-specific questions
Helpful links for rare disease information and support
Clinical profile data for this condition is not yet available. Phenotype information may still be loading below.
Research studies investigating treatments and therapies for this condition.
Active Trials
Total Trials
Data from ClinicalTrials.gov Jan 30, 2026
Consider asking your healthcare providers these condition-specific questions
European rare disease database
Genetic and Rare Diseases Info Center
AI-Generated Content: This summary was generated using AI. Content has been fact-checked. Always consult with qualified healthcare providers for medical guidance.
Organizations with approved therapies for this disease
Kisho delivers this disease record via API, including phenotypes (HPO), genes, orphan drug designations, screening status, and PAG mapping, with version history and governance.