Aplastic Anemia | UniteRare Disease Library

Overview

Aplastic Anemia is treated with 1 medication in our database, including COSELA. 1 of these has manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by G1. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Aplastic Anemia treatment below.

Inheritance

Variable

Can be inherited in different ways depending on the underlying gene

Age of Onset

Variable

Can begin at different ages, from infancy through adulthood

NORD ↗

FDA & Trial Timeline

10 events

Aug 2026CK0801 for Treatment of Aplastic Anemia

Cellenkos, Inc. — PHASE2

TrialNOT YET RECRUITING

Aug 2026UI-Romi-02; Romiplostim Added to Standard of Care for Treatment Naive and Relapsed or Refractory Severe Aplastic Anemia

Anjali Sharathkumar — PHASE2

TrialNOT YET RECRUITING

Mar 2026The HOPE Biobank Resource (BMT CTN 2402 HOPE)

Medical College of Wisconsin

TrialNOT YET RECRUITING

Feb 2026Adverse Effects of ATG/ALG Therapy in Aplastic Anemia

Institute of Hematology & Blood Diseases Hospital, China

TrialRECRUITING

Jan 2026Romiplostim Versus rhTPO for Platelet Engraftment After Transplant in MDS and AA

The First Affiliated Hospital of Soochow University — PHASE2

TrialNOT YET RECRUITING

Jan 2026A Study of Romiplostim N01 Plus IST vs. Placebo Plus IST for Treatment-Naive Severe Aplastic Anemia

Qilu Pharmaceutical Co., Ltd. — PHASE3

TrialNOT YET RECRUITING

Dec 2025Safety and Efficacy of the JAK1 Inhibitor Combined With Intensive Immunosuppressive Therapy in Severe Aplastic Anemia

Institute of Hematology & Blood Diseases Hospital, China — PHASE1, PHASE2

TrialNOT YET RECRUITING

Dec 2025The Safety and Efficacy of Daratumumab in Patients With Refractory Aplastic Anemia

Institute of Hematology & Blood Diseases Hospital, China — PHASE1, PHASE2

TrialNOT YET RECRUITING

Dec 2025Clinico-Epidemiological Profile Of Acquired Aplastic Anemia Among Children At Assiut Governorate .

Assiut University

TrialNOT YET RECRUITING

Nov 2025A Prediction Model in Pregnant Women With Aplastic Anemia

Peking University People's Hospital — NA

TrialRECRUITING

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

Treatments

1 available

COSELA

trilaciclib· G1

Detailed copay and financial assistance information is not publicly available for this medication at this time. Please consult your pharmacist or the manufacturer's official patient support program fo

View Details →Copay Card ↗Patient Assistance ↗

Clinical Trials

20 recruitingView all trials with filters →

Phase 31 trial

A Trial Comparing Unrelated Donor BMT With IST for Pediatric and Young Adult Patients With Severe Aplastic Anemia (TransIT, BMT CTN 2202)

Phase 3

Active

PI: David Williams, MD (Boston Children's Hospital) · Sites: Birmingham, Alabama; Phoenix, Arizona +50 more · Age: 0–25 yrs

Phase 213 trials

Exercise Training and NR Supplementation Trial to Improve Fitness in AYA HCT Survivors

Phase 2

Actively Recruiting

PI: Sogol Mostoufi-Moab, MD, MSCE (Children's Hospital of Philadelphia) · Sites: Duarte, California; Philadelphia, Pennsylvania +1 more · Age: 10–30 yrs

A Study of Emapalumab for Pediatric Aplastic Anemia

Phase 2

Actively Recruiting

PI: Andromachi Scaradavou, MD (Memorial Sloan Kettering Cancer Center) · Sites: New York, New York; Cincinnati, Ohio +4 more · Age: 0–25 yrs

Efficacy and Safety of Sirolimus With or Without Cyclosporin A in Chinese Patients With Aplastic Anemia Refractory/Intolerant to Cyclosporin A

Phase 2

Actively Recruiting

· Sites: Beijing, Beijing Municipality · Age: 18–99 yrs

MT2023-20: Hematopoietic Cell Transplant With Reduced Intensity Conditioning and Post-transplant Cyclophosphamide for Severe Aplastic Anemia and Other Forms of Acquired Bone Marrow Failure.

Phase 2

Actively Recruiting Prior treatment eligible

PI: Christen Ebens, MD, MPH · Sites: Minneapolis, Minnesota · Age: 0–75 yrs

Methylprednisolone, Horse Anti-Thymocyte Globulin, Cyclosporine, Filgrastim, and/or Pegfilgrastim or Pegfilgrastim Biosimilar in Treating Patients With Aplastic Anemia or Low or Intermediate-Risk Myelodysplastic Syndrome

Phase 2

Actively Recruiting Prior treatment eligible

PI: Tapan M Kadia (M.D. Anderson Cancer Center) · Sites: Houston, Texas · Age: 18–99 yrs

Dose-attenuated IST and Hetrombopag in Elderly (≥65 Years) Patients With Severe Aplastic Anemia

Phase 2

Actively Recruiting Prior treatment eligible

PI: Jun Shi, PhD (Institute of Hematology & Blood Diseases Hospital,) · Sites: Tianjin, Tianjin Municipality · Age: 65–99 yrs

Clinical Trial of Upfront Haploidentical or Unrelated Donor BMT to Restore Normal Hematopoiesis in Aplastic Anemia

Phase 2

Actively Recruiting Prior treatment eligible

PI: Jennifer Romeril · Sites: Birmingham, Alabama; Duarte, California +18 more · Age: 3–75 yrs

Haploidentical Bone Marrow Transplant With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia

Phase 2

Actively Recruiting Prior treatment eligible

PI: Melhem Solh, MD (Blood and Marrow Transplant Group of Georgia) · Sites: Atlanta, Georgia; Atlanta, Georgia · Age: 1–75 yrs

Standard Immunosuppressive Therapy Combined With Romiplostim N01 as First-line Treatment for Severe Aplastic Anemia

Phase 2

Actively Recruiting

PI: Jun Shi, PhD (Institute of Hematology & Blood Diseases Hospital,) · Sites: Tianjin, Tianjin Municipality · Age: 12–99 yrs

Clinical Study on Modified Allogeneic Hematopoietic Stem Cell Transplantation Regimen for Severe Aplastic Anemia

Phase 2

Actively Recruiting

PI: wang PI sanbin, Doctor · Sites: Kunming, Yunnan · Age: 3–65 yrs

Haploidentical HCT for Severe Aplastic Anemia

Phase 2

Actively Recruiting Prior treatment eligible

PI: Amr Qudeimat, MD (St. Jude Children's Research Hospital) · Sites: Memphis, Tennessee · Age: 0–21 yrs

Ruxolitinib Based GVHD Prophylaxis Regimen Before, During, and After Hematopoietic Cell Transplantation in Older Adult Patients With Acquired Aplastic Anemia

Phase 2

Actively Recruiting Prior treatment eligible

PI: Rachel B. Salit, MD (Fred Hutch/University of Washington Cancer Consort) · Sites: Seattle, Washington · Age: 18–99 yrs

Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis

Phase 2

Actively Recruiting Prior treatment eligible

PI: Richard W Childs, M.D. (National Heart, Lung, and Blood Institute (NHLBI)) · Sites: Bethesda, Maryland · Age: 4–75 yrs

Other6 trials

Adverse Effects of ATG/ALG Therapy in Aplastic Anemia

Actively Recruiting

· Sites: Tianjin

RACE 2: a Long Term Follow-up of Patients Participating in the RACE Trial

Enrolling by Invitation

PI: Regis Peffault de Latour, Prof, MD (Hospital St. Louis, Paris, France) · Sites: Paris

Umbilical Cord Blood Transplantation From Unrelated Donors

Actively Recruiting

PI: Omar Aljitawi, MD (Professor - Department of Medicine, Hematology/Onc) · Sites: Rochester, New York · Age: 0–75 yrs

Multi-omics Profiling of Patients With Aplastic Anemia Before and After CD7-CAR-T Therapy

Active

PI: Hai Cheng (The Affiliated Hospital oh Xuzhou Medical Universi) · Sites: Xuzhou, Jiangsu · Age: 18–70 yrs

Revolade Tablets Specified Drug-use Survey

Actively Recruiting

PI: Novartis Pharmaceuticals (Novartis Pharmaceuticals) · Sites: Nagoya, Aichi-ken; Nagoya, Aichi-ken +15 more · Age: 6–17 yrs

A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)

Actively Recruiting

PI: Heather Stefanski, MD, PhD (NMDP/CIBMTR) · Sites: Birmingham, Alabama; Gilbert, Arizona +140 more

Specialists

Showing 25 of 110View all specialists →

HY

Hirohito Yamazaki

Kanazawa, Ishikawa-ken

Specialist

Rare Disease Specialist

31 Aplastic Anemia publications

Overview

FDA & Trial Timeline

Treatments

Clinical Trials

Specialists

Treatment Centers

Travel Grants

Community

Latest news about Aplastic Anemia

Caregiver Resources

Common questions about Aplastic Anemia