Overview
Orphanet code 506219 refers to a rare disorder that may be considered for treatment with a hematopoietic stem cell transplant (HSCT), also known as a bone marrow transplant. This category covers a group of serious conditions where the blood-forming cells in the bone marrow do not work properly, or where the immune system is severely affected. Because Orphanet code 506219 is a broad grouping rather than a single named disease, the exact symptoms and features depend on the specific underlying condition a person has been diagnosed with. In general, diseases in this category can affect the production of red blood cells, white blood cells, or platelets, leading to problems like anemia, frequent infections, or abnormal bleeding. Some conditions in this group also affect the immune system directly, making it very hard for the body to fight off germs. Hematopoietic stem cell transplant is considered when other treatments have not worked or when the disease is severe enough that replacing the bone marrow offers the best chance of a cure or long-term improvement. Treatment planning is highly individualized. A transplant team, including specialists in blood diseases and immunology, will assess whether a patient is a good candidate for HSCT based on their specific diagnosis, overall health, and availability of a matched donor. Supportive care, such as blood transfusions, antibiotics, and growth factors, is often used alongside or before transplant.
Key symptoms:
Severe or repeated infections that are hard to treatUnusual tiredness and weakness due to low red blood cell counts (anemia)Easy bruising or bleeding that does not stop quicklyPale skin or yellowing of the skinSwollen lymph nodes or enlarged spleenFever without a clear causeSlow growth or failure to thrive in childrenBone pain or joint painShortness of breath during normal activities
Variable
Can be inherited in different ways depending on the underlying gene
Variable
Can begin at different ages, from infancy through adulthood
FDA & Trial Timeline
2 eventsOmisirge: FDA approved
for use in adults and pediatric patients 12 years and older with hematologic malignancies who are planned for umbilical cord blood transplantation following myeloablative conditioning to reduce the time to neutrophil recovery and the incidence of infection
Mozobil (r): FDA approved
To enhance mobilization of hematopoietic stem cells to the peripheral blood for collection and subsequent autologous transplantation in patients with lymphoma and multiple myeloma
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
1 availableOmisirge
for use in adults and pediatric patients 12 years and older with hematologic malignancies who are planned for umbilical cord blood transplantation following myeloablative conditioning to reduce the ti…
for use in adults and pediatric patients 12 years and older with hematologic malignancies who are planned for umbilical cord blood transplantation following myeloablative conditioning to reduce the time to neutrophil recovery and the incidence of infection
Clinical Trials
View all trials with filters →No actively recruiting trials found for Rare disorder potentially indicated for hematopoietic stem cell transplant at this time.
New trials open frequently. Follow this disease to get notified.
Specialists
View all specialists →No specialists are currently listed for Rare disorder potentially indicated for hematopoietic stem cell transplant.
Treatment Centers
8 centersBaylor College of Medicine Rare Disease Center ↗
Baylor College of Medicine
📍 Houston, TX
🏥 NORDStanford Medicine Rare Disease Center ↗
Stanford Medicine
📍 Stanford, CA
🔬 UDNNIH Clinical Center Undiagnosed Diseases Program ↗
National Institutes of Health
📍 Bethesda, MD
🔬 UDNUCLA UDN Clinical Site ↗
UCLA Health
📍 Los Angeles, CA
🔬 UDNBaylor College of Medicine UDN Clinical Site ↗
Baylor College of Medicine
📍 Houston, TX
🔬 UDNHarvard/MGH UDN Clinical Site ↗
Massachusetts General Hospital
📍 Boston, MA
🏥 NORDMayo Clinic Center for Individualized Medicine ↗
Mayo Clinic
📍 Rochester, MN
👤 Mayo Clinic Center for Individualized Medicine
🏥 NORDUCLA Rare Disease Day Program ↗
UCLA Health
📍 Los Angeles, CA
Travel Grants
No travel grants are currently matched to Rare disorder potentially indicated for hematopoietic stem cell transplant.
Community
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Caregiver Resources
NORD Caregiver Resources
Support, advocacy, and financial assistance for caregivers of rare disease patients.
Mental Health Support
Rare disease caregiving can be isolating. Connect with counseling and peer support.
Family & Caregiver Grants
Financial assistance programs specifically for caregivers of rare disease patients.
Social Security Disability
Learn how rare disease patients may qualify for SSDI/SSI benefits.
Questions for your doctor
Bring these to your next appointment
- Q1.What is my specific diagnosis, and how does it relate to the need for a bone marrow transplant?,How urgently do I need a transplant, and what happens if we wait?,How do we find a matched donor, and what are my options if no perfect match is found?,What are the main risks of the transplant, and how will you manage them?,Are there any clinical trials or newer treatments I should know about?,What supportive treatments will I need before and after the transplant?,What does long-term follow-up look like after a successful transplant?
Common questions about Rare disorder potentially indicated for hematopoietic stem cell transplant
What is Rare disorder potentially indicated for hematopoietic stem cell transplant?
Orphanet code 506219 refers to a rare disorder that may be considered for treatment with a hematopoietic stem cell transplant (HSCT), also known as a bone marrow transplant. This category covers a group of serious conditions where the blood-forming cells in the bone marrow do not work properly, or where the immune system is severely affected. Because Orphanet code 506219 is a broad grouping rather than a single named disease, the exact symptoms and features depend on the specific underlying condition a person has been diagnosed with. In general, diseases in this category can affect the produc
What treatment and support options exist for Rare disorder potentially indicated for hematopoietic stem cell transplant?
1 patient support program are currently tracked on UniteRare for Rare disorder potentially indicated for hematopoietic stem cell transplant. See the treatments and support programs sections for copay assistance, eligibility, and contact details.