Overview
Orphanet code 565779 refers to a rare disorder that is either a condition for which organ or stem cell transplantation may be considered as a treatment, or a complication that arises after a transplant has been performed. This broad category covers a wide range of serious conditions where the body's organs or immune system are severely affected — sometimes to the point where replacing a failing organ (such as the liver, kidney, heart, or bone marrow) becomes necessary to save a person's life. After a transplant, the body can face new challenges. The immune system may attack the new organ (a process called rejection), or the new immune cells from a donor may attack the recipient's body (known as graft-versus-host disease, or GVHD). Infections, organ dysfunction, and medication side effects are also common concerns in the post-transplant period. Because this Orphanet code covers a group of conditions rather than a single disease, symptoms, treatments, and outcomes vary widely depending on the specific underlying disorder. Management typically involves a specialized transplant team, immunosuppressive medications to prevent rejection, and close long-term monitoring. Early identification of complications is critical to improving outcomes for patients in this category.
Key symptoms:
Organ failure or severely reduced organ functionExtreme fatigue and weaknessFrequent or severe infectionsSkin rash or blistering (possible sign of graft-versus-host disease)Jaundice (yellowing of the skin or eyes)Nausea, vomiting, or diarrheaSwelling in the legs or abdomenShortness of breathFever without a clear causeUnexplained weight lossBleeding or bruising easilyConfusion or changes in mental alertness
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
6 eventsGIANCARLO FATOBENE — PHASE2
Sichuan University — PHASE2
Henry Ford Health System — PHASE1
Hospital Israelita Albert Einstein — PHASE2, PHASE3
Busulfex: FDA approved
For use in combination with cyclophosphamide as a conditioning regimen prior to allogeneic hematopoietic progenitor cell transplantation for chronic myelogenous leukemia.
Digibind: FDA approved
Treatment of potentially life threatening digitalis intoxication in patients who are refractory to management by conventional therapy.
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
2 availableBusulfex
For use in combination with cyclophosphamide as a conditioning regimen prior to allogeneic hematopoietic progenitor cell transplantation for chronic myelogenous leukemia.
Digibind
Specialists
View all specialists →No specialists are currently listed for Rare disorder potentially indicated for transplant or complication after transplantation.
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 transplant or complication after transplantation.
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 specific condition do I have, and why is transplant being considered for me?,What type of transplant would I need, and how do I get on the waiting list?,What are the most likely complications I should watch for after transplant?,What medications will I need to take long-term, and what are their side effects?,How will this condition and the transplant affect my daily life, work, or school?,Are there clinical trials or newer treatments I should know about?,What support services or patient organizations are available to help me and my family?
Common questions about Rare disorder potentially indicated for transplant or complication after transplantation
What is Rare disorder potentially indicated for transplant or complication after transplantation?
Orphanet code 565779 refers to a rare disorder that is either a condition for which organ or stem cell transplantation may be considered as a treatment, or a complication that arises after a transplant has been performed. This broad category covers a wide range of serious conditions where the body's organs or immune system are severely affected — sometimes to the point where replacing a failing organ (such as the liver, kidney, heart, or bone marrow) becomes necessary to save a person's life. After a transplant, the body can face new challenges. The immune system may attack the new organ (a p
Are there clinical trials for Rare disorder potentially indicated for transplant or complication after transplantation?
Yes — 2 recruiting clinical trials are currently listed for Rare disorder potentially indicated for transplant or complication after transplantation on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.
What treatment and support options exist for Rare disorder potentially indicated for transplant or complication after transplantation?
2 patient support programs are currently tracked on UniteRare for Rare disorder potentially indicated for transplant or complication after transplantation. See the treatments and support programs sections for copay assistance, eligibility, and contact details.