Comprehensive, easy-to-understand information about this condition
How we create this content →Research studies investigating treatments and therapies for this condition.
Active Trials
Total Trials
Data from ClinicalTrials.gov Jan 31, 2026
Research studies investigating treatments and therapies for this condition.
Active Trials
Total Trials
Data from ClinicalTrials.gov Jan 31, 2026
The limited documentation surrounding this syndrome is primarily due to its extreme rarity, affecting fewer than 1 in 1,000,000 individuals. This scarcity makes it challenging to conduct systematic clinical studies and gather comprehensive data on its clinical features and management. As research progresses, it is hoped that more information will become available to better understand and treat this condition.
To navigate your care effectively, consider seeking a hematologist with expertise in autoimmune disorders, particularly those involving hemolytic anemia and thrombocytopenia. While there are currently no identified patient organizations, you can find resources and support through the Genetic and Rare Diseases Information Center (GARD) at rarediseases.info.nih.gov. Participating in clinical trials may also provide access to cutting-edge treatments and contribute to research efforts. Keep an eye on ongoing studies that might be relevant to your condition.
There are currently three orphan drugs designated for this condition, including a humanized IgG4 monoclonal antibody against total complement component 1, subcomponent s, Mezagitamab, and rituximab. Additionally, there are three active clinical trials exploring potential treatments. For more information on these trials, you can visit the ClinicalTrials.gov search page: https://clinicaltrials.gov/search?cond=autoimmune%20hemolytic%20anemia-autoimmune%20thrombocytopenia-primary%20immunodeficiency%20syndrome.
Actionable guidance for navigating care for autoimmune hemolytic anemia-autoimmune thrombocytopenia-primary immunodeficiency syndrome
To navigate your care effectively, consider seeking a hematologist with expertise in autoimmune disorders, particularly those involving hemolytic anemia and thrombocytopenia. While there are currently no identified patient organizations, you can find resources and support through the Genetic and Rare Diseases Information Center (GARD) at rarediseases.info.nih.gov. Participating in clinical trials may also provide access to cutting-edge treatments and contribute to research efforts. Keep an eye on ongoing studies that might be relevant to your condition.
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 31, 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.
Kisho delivers this disease record via API, including phenotypes (HPO), genes, orphan drug designations, screening status, and PAG mapping, with version history and governance.