Anemia resulting from bone marrow failure (aplastic or hypoplastic bone marrow). The production of erythroblasts and red cells is markedly decreased, and it may be associated with decreased production...
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 Feb 8, 2026
Research studies investigating treatments and therapies for this condition.
Active Trials
Total Trials
Data from ClinicalTrials.gov Feb 8, 2026
The documentation for aplastic anemia is limited due to its relatively rare occurrence and the complexity of its causes, which can be idiopathic or secondary to environmental factors. This rarity makes large-scale clinical studies challenging, resulting in less comprehensive data. Additionally, the genetic basis is still being explored, which contributes to the gaps in understanding the disease fully.
The primary clinical feature of aplastic anemia is a reduction in blood cell production, leading to symptoms of anemia, which can include fatigue, pallor, and shortness of breath. Due to decreased granulocytes, patients may also experience increased susceptibility to infections. Thrombocytopenia can result in easy bruising or bleeding. These symptoms are often present from the onset of the condition, although the severity can vary among individuals.
To navigate your journey with aplastic anemia, consider seeking a hematologist with expertise in bone marrow disorders. They can provide specialized care and management strategies tailored to your needs. Additionally, resources such as the Aplastic Anemia & MDS International Foundation (https://aamds.org) and the Aplastic Anemia & Myelodysplasia Association of Canada (https://aamac.ca) can offer support and information. Participation in patient registries or natural history studies may also be beneficial for gaining insights into the condition and connecting with others affected by it.
Currently, there are 122 active clinical trials exploring various aspects of aplastic anemia, including potential treatments and management strategies. While there are no orphan drugs specifically designated for this condition, the ongoing trials may offer insights into new therapeutic options. You can explore these trials at ClinicalTrials.gov by searching for 'aplastic anemia' to find studies that may be relevant to your situation.
Actionable guidance for navigating care for aplastic anemia
To navigate your journey with aplastic anemia, consider seeking a hematologist with expertise in bone marrow disorders. They can provide specialized care and management strategies tailored to your needs. Additionally, resources such as the Aplastic Anemia & MDS International Foundation (https://aamds.org) and the Aplastic Anemia & Myelodysplasia Association of Canada (https://aamac.ca) can offer support and information. Participation in patient registries or natural history studies may also be beneficial for gaining insights into the condition and connecting with others affected by it.
Consider asking your healthcare providers these condition-specific questions
Connect with organizations supporting the aplastic anemia community
Helpful links for rare disease information and support
Research studies investigating treatments and therapies for this condition.
Active Trials
Total Trials
Data from ClinicalTrials.gov Feb 8, 2026
Patient Advocacy Groups (PAGs) provide support, resources, and community for patients and caregivers.
Consider asking your healthcare providers these condition-specific questions
Online Mendelian Inheritance in Man
European rare disease database
Genetic and Rare Diseases Info Center
2 peer-reviewed sources from PubMed
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
No approved therapies yet
Research is ongoing — 7 companies have orphan drug designations 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.