Acute myeloid leukemia and myelodysplastic syndromes related to radiation

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ORPHA:164726D46.7
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Overview

Acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) related to radiation are therapy-related or exposure-related hematologic malignancies that develop as a consequence of prior exposure to ionizing radiation. This condition is classified under therapy-related myeloid neoplasms and is recognized by Orphanet under code 164726 (ICD-10: D46.7). These disorders arise when radiation damages the DNA of hematopoietic stem cells in the bone marrow, leading to genomic instability, abnormal blood cell production, and eventual malignant transformation. The latency period between radiation exposure and disease onset is typically 2 to 10 years, though it can vary depending on the dose and type of radiation received. The disease primarily affects the hematopoietic (blood-forming) system. In MDS, the bone marrow produces abnormal and ineffective blood cells, leading to cytopenias — low red blood cells (anemia), low white blood cells (leukopenia), and low platelets (thrombocytopenia). Patients may experience fatigue, recurrent infections, easy bruising, and bleeding. MDS related to radiation frequently progresses to AML, a rapidly progressing cancer of the blood and bone marrow characterized by the accumulation of immature myeloid blast cells. Radiation-related AML often involves complex cytogenetic abnormalities, including deletions of chromosomes 5 and 7, which are associated with a poor prognosis. Treatment options are generally similar to those for de novo AML and MDS but outcomes tend to be less favorable. Management may include supportive care (transfusions, growth factors, antibiotics), hypomethylating agents such as azacitidine or decitabine, intensive chemotherapy, and allogeneic hematopoietic stem cell transplantation, which remains the only potentially curative approach. However, therapy-related myeloid neoplasms, including those caused by radiation, are generally associated with poorer response rates to standard chemotherapy and lower overall survival compared to primary AML or MDS. Clinical trials exploring novel targeted therapies and combination regimens are ongoing.

Also known as:

Inheritance

Sporadic

Usually appears on its own, not inherited from a parent

Age of Onset

Adult

Begins in adulthood (age 18 or older)

Orphanet ↗NORD ↗

FDA & Trial Timeline

1 event
Jan 2005

ThyroShield: FDA approved

For use as a thyroid blocking agent in pediatric patients exposed to radiactive iodine

FDAcompleted

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

1 available

Reblozyl

LUSPATERCEPT· Celgene Corporation
REBLOZYL is indicated for the treatment of anemia without previous erythropoiesis stimulating agent use (ESA-naïve) in adult patients with very low- to intermediate-risk myelodysplastic syndromes (MDS

REBLOZYL is indicated for the treatment of anemia without previous erythropoiesis stimulating agent use (ESA-naïve) in adult patients with very low- to intermediate-risk myelodysplastic syndromes (MDS) who may require regular red blood cell (RBC) transfusions

No actively recruiting trials found for Acute myeloid leukemia and myelodysplastic syndromes related to radiation at this time.

New trials open frequently. Follow this disease to get notified.

Search ClinicalTrials.gov ↗Join the Acute myeloid leukemia and myelodysplastic syndromes related to radiation community →

Specialists

2 foundView all specialists →
AO
Amanda Olson
Specialist
PI on 2 active trials19 Acute myeloid leukemia and myelodysplastic syndromes related to radiation publications
NK
Nancy A. Kernan
NEW YORK, NY
Specialist
PI on 1 active trial

Treatment Centers

8 centers
🏥 NORD

Baylor College of Medicine Rare Disease Center

Baylor College of Medicine

📍 Houston, TX

🏥 NORD

Stanford Medicine Rare Disease Center

Stanford Medicine

📍 Stanford, CA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

🔬 UDN

UCLA UDN Clinical Site

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🏥 NORD

UCLA Rare Disease Day Program

UCLA Health

📍 Los Angeles, CA

Financial Resources

3 resources
Reblozyl(LUSPATERCEPT)Celgene Corporation

ThyroShield

Fleming & Company, Pharmaceuticals

Unverified — confirm before calling
copay card
copay assistancePatient Assistance
Accepting applications

AZACITIDINE

Pilnova Pharma Inc

Myelodysplastic Syndrome

Unverified — confirm before calling
copay card
Copay CardPatient Assistance
Accepting applications

Travel Grants

No travel grants are currently matched to Acute myeloid leukemia and myelodysplastic syndromes related to radiation.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Acute myeloid leukemia and myelodysplastic syndromes related to radiation

2 articles
Clinical trialUNITERAREApr 5, 2026
New Recruiting Trial: Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms
Researchers are testing a new drug called pacritinib in people ages 12 and older who have myelodysplastic syndromes or myelodysplastic/myeloproliferative neopla
Clinical trialUNITERAREApr 3, 2026
New Recruiting Trial: A Study to Compare Elritercept With Epoetin Alfa to Treat Anemia in Adults With Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS) Who Need Regular Blood Transfusions
Researchers are testing a new drug called elritercept to see if it works better than a standard treatment (epoetin alfa) for anemia in adults with myelodysplast
See all news about Acute myeloid leukemia and myelodysplastic syndromes related to radiation

Caregiver Resources

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Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

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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.

Common questions about Acute myeloid leukemia and myelodysplastic syndromes related to radiation

What is Acute myeloid leukemia and myelodysplastic syndromes related to radiation?

Acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) related to radiation are therapy-related or exposure-related hematologic malignancies that develop as a consequence of prior exposure to ionizing radiation. This condition is classified under therapy-related myeloid neoplasms and is recognized by Orphanet under code 164726 (ICD-10: D46.7). These disorders arise when radiation damages the DNA of hematopoietic stem cells in the bone marrow, leading to genomic instability, abnormal blood cell production, and eventual malignant transformation. The latency period between radiation exp

How is Acute myeloid leukemia and myelodysplastic syndromes related to radiation inherited?

Acute myeloid leukemia and myelodysplastic syndromes related to radiation follows a sporadic inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Acute myeloid leukemia and myelodysplastic syndromes related to radiation typically begin?

Typical onset of Acute myeloid leukemia and myelodysplastic syndromes related to radiation is adult. Age of onset can vary across affected individuals.

Which specialists treat Acute myeloid leukemia and myelodysplastic syndromes related to radiation?

2 specialists and care centers treating Acute myeloid leukemia and myelodysplastic syndromes related to radiation are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.

What treatment and support options exist for Acute myeloid leukemia and myelodysplastic syndromes related to radiation?

2 patient support programs are currently tracked on UniteRare for Acute myeloid leukemia and myelodysplastic syndromes related to radiation. See the treatments and support programs sections for copay assistance, eligibility, and contact details.