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Home › Conditions › severe aplastic anemia

Severe aplastic anemia

Part of Anemia

5 published articles · Updated continuously

Clinical Trial Landscape

Clinical Trials for severe aplastic anemia

35 trials tracked for severe aplastic anemia: 2 in phase 3 or 4 and 2 with published results. The most-cited published study has 151 citations.

35Trials tracked
2Phase 3 & 4
0Recruiting
2With published results
Phase distribution
Phase 3 2 Phase 2 28 Other / NA 5
  1. Phase 3 Stem Cell Transplant (SCT) for Dyskeratosis Congenita or SAA Completed · 151 cited
  2. Phase 3 A Study to Assess Efficacy and Safety of PF-06462700 in Japanese Participants With Aplastic Anemia Completed · 1 cited
  3. Phase 2 A Pilot Study of Fludarabine Plus Cyclophosphamide in Refractory Severe Aplastic Anemia Completed
  4. Phase 2 Cyclophosphamide and Anti-thymocyte Globulin Followed By Methotrexate and Cyclosporine in Preventing Chronic Graft-Versus-Host Disease in Patients With Severe Aplastic Anemia Undergoing Donor Bone Marrow Transplant Completed
  5. Phase 2 A Two-Step Approach to Reduced Intensity Bone Marrow Transplant for Patients With Hematological Malignancies Completed
  6. Phase 2 NMA Allogeneic Hematopoietic Cell Transplant in Hematologic Cancer/Disorders Completed
Show 29 more trials
  1. Phase 2 Transplantation of Partially Mismatched Related or Matched Unrelated Bone Marrow for Patients With Refractory Severe Aplastic Anemia Completed
  2. Phase 2 Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies Completed
  3. Phase 2 Post Transplant Cyclophosphamide (Cytoxan) for GvHD Prophylaxis Completed
  4. Phase 2 Eltrombopag Combined With Cyclosporine as First Line Therapy in Patients With Severe Acquired Aplastic Anemia Completed
  5. Phase 2 Eltrombopag in Combination With Rabbit Anti-thymocyte Globulin/Cyclosporine A in Naive Aplastic Anemia (AA) Subjects Completed
  6. Phase 2 Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure Syndromes Completed
  7. Phase 2 Posaconazole Prophylaxis During ATG Treatment for hMDS/AA Patients Completed
  8. Phase 2 Cyclophosphamide Plus Cyclosporine in Treatment-Naive Severe Aplastic Anemia Completed
  9. Phase 2 Thymoglobulin and Cyclosporine in Patients With Aplastic Anemia or Myelodysplastic Syndrome Completed
  10. Phase 2 A Phase 2 Study to Evaluate the Efficacy and Safety of AMG531 in Aplastic Anemia Completed
  11. Phase 2 Unrelated Cord Blood Transplant Plus a Haplo-Identical (Half-Matched), T-Cell Depleted Stem Transplant From a Related Donor for Subjects With High Risk Malignancies Completed
  12. Phase 2 Efficacy and Safety of Eltrombopag In Patients With Severe and Very Severe Aplastic Anemia Completed
  13. Phase 2 Reduced-Intensity Conditioning Before Donor Stem Cell Transplant in With High-Risk Hematologic Malignancies Completed
  14. Phase 2 NMA Haplo or MUD BMT for Newly Diagnosed Severe Aplastic Anemia Completed
  15. Phase 2 Study Assessed the Safety and Efficacy of Eltrombopag in Chinese Refractory or Relapsed Severe Aplastic Anemia (SAA) Subjects. Completed
  16. Phase 2 Optimizing Haploidentical Aplastic Anemia Transplantation (BMT CTN 1502) Completed
  17. Phase 2 Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine Completed
  18. Phase 2 Bone Marrow Transplantation of Patients in Remission Using Partially Matched Relative Donor Completed
  19. Phase 2 Reduced Intensity Chemotherapy and Radiation Therapy Before Donor Stem Cell Transplant in Treating Patients With Hematologic Malignancies Completed
  20. Phase 2 Anti-thymocyte Globulin and Cyclosporine as First-Line Therapy in Treating Patients With Severe Aplastic Anemia Completed
  21. Phase 2 Rabbit Antithymocyte Globulin Versus Campath-1H for Treating Severe Aplastic Anemia Completed
  22. Phase 2 Eltrombopag for Moderate Aplastic Anemia Completed
  23. Phase 2 Rabbit Antithymocyte Globulin (Thymoglobuline) With Ciclosporin for Patients With Acquired Aplastic Anaemia Completed
  24. Phase 2 Danazol for Genetic Bone Marrow and Lung Disorders Completed
  25. N/A Purine Analog-Based Conditioning in Patients With Severe Aplastic Anemia Completed
  26. N/A Unrelated Umbilical Cord Blood (UBC)Transplantation Completed
  27. N/A Fludarabine Phosphate, Melphalan, Total-Body Irradiation, Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer or Bone Marrow Failure Disorders Completed
  28. N/A Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia Completed
  29. N/A Fludarabine Based Conditioning for Allogeneic Transplantation for Advanced Hematologic Malignancies Completed

Showing the 35 most-cited and recently-updated of 35 trials. Browse the full registry →

Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.

What the trials found Updated — new results For clinicians

Severe aplastic anemia: what the trials found

Several trials have evaluated eltrombopag in severe aplastic anemia. In a phase 2 trial (N=20), eltrombopag achieved a hematologic response rate of 70.0% at 6 months 6. Another phase 2 trial (N=54) reported overall hematologic response rates of 46.3% at 6 months, 40.7% at 3 months, and 18.5% at both 12 and 24 months 8. A smaller phase 2 trial (N=10) found that eltrombopag led to complete or partial response in 2 patients at 3 months, and 1 complete response at 6 months, with a mean exposure of 493.7 days 16. Additional phase 2 trials (N=34 and N=13) also investigated eltrombopag 1720.

Fludarabine-based regimens have been studied in multiple phase 2 trials. Trials NCT01384513 (N=40), NCT01760655 (N=62), NCT02566304 (N=35), and NCT01529827 (N=94) all evaluated fludarabine or fludarabine phosphate 14521. In a phase 3 trial (N=36), Campath 1H was associated with incidences of regimen-related mortality at 100 days (1 vs 2), grade 2-4 acute GVHD (1 vs 0), and grade 3-4 acute GVHD (0 vs 0) 2. Thymoglobulin was evaluated in two phase 2 trials (N=18 and N=21) 910; in one, 17 participants developed grade II-IV or III-IV acute GVHD 9. Cyclophosphamide was studied in a phase 2 trial (N=39), with 30 cases of grade II-IV acute GVHD 12. A phase 3 trial (N=3) of PF-06462700 reported transfusion independence at weeks 12 and 24 in 0 and 2 participants, respectively 3.

Recent results — preliminary, needs further review

  • A phase 2 trial (N=20) of posaconazole is unconfirmed 7.
  • A phase 2 trial (N=32) of antithymocyte globulin (ATG) reported neutrophil recovery in 93.5%, platelet recovery in 77.4%, and graft-failure-free survival in 77.4% 11.
  • A phase 2 trial (N=35) of rabbit antithymocyte globulin is unconfirmed 13.
  • A phase 2 trial (N=35) of AMG531 is unconfirmed 14.
  • A phase 2 trial (N=41) of anti-thymocyte globulin is unconfirmed 15.

For the clinician treating this condition

  • Eltrombopag demonstrates hematologic response in severe aplastic anemia, with response rates varying by time point (e.g., 70% at 6 months in one trial) 6816.
  • Fludarabine-based regimens have been evaluated in multiple phase 2 trials, though specific outcome data are not provided 14521.
  • Campath 1H, thymoglobulin, and cyclophosphamide have been studied in the context of GVHD and regimen-related mortality, with variable incidences 2912.

AI synthesis of 19 cited trials, updated Jun 29, 2026. Informational only — not medical advice; trial data sourced from ClinicalTrials.gov. How we use AI.

HCP Mode — summaries include clinical detail, trial data, and statistical outcomes.
Patient Mode — summaries use plain language, avoiding clinical jargon.
Isolated gastric Kaposi sarcoma occurs following allogeneic hematopoietic stem cell transplantation in rare cases
Rheumatology Sys. Review
Isolated gastric Kaposi sarcoma occurs following allogeneic hematopoietic stem cell transplantation in rare cases Rare Gastric Kaposi Sarcoma Found After Stem Cell Transplant
This case report and literature review identifies 20 cases of Kaposi sarcoma following allogeneic hematopoietic stem cell transplantation. T…
A rare case of stomach cancer linked to stem cell transplants highlights the need for early testing when patients have new digestive symptom…
Frontiers Sep 18, 2026
Eltrombopag addition alters clonal hematopoiesis dynamics in untreated severe aplastic anemia patients over 24 months
Drug Pipeline RCT
Eltrombopag addition alters clonal hematopoiesis dynamics in untreated severe aplastic anemia patients over 24 months Adding Eltrombopag helps severe anemia patients recover blood counts significantly faster
This Phase 3 randomized trial reviewed patient samples from 170 untreated patients with severe or very severe aplastic anemia receiving eltr…
Adding Eltrombopag helps severe anemia patients recover blood counts faster without increasing cancer risks but genetic changes rise over ti…
NEJM Evid Apr 28, 2026
Eltrombopag plus immunosuppression for severe aplastic anemia shows clonal evolution patterns
Pediatrics Phase II
Eltrombopag plus immunosuppression for severe aplastic anemia shows clonal evolution patterns Treatment for severe anemia reveals two paths to cancer risk
This phase 2 observational study followed 204 patients with severe aplastic anemia treated with immunosuppression plus eltrombopag for a med…
New research tracks cancer risks in severe anemia patients treated with standard therapy over five years
NEJM Evid Apr 28, 2026
Pretreatment sTNFR1 and infection predict transplant-associated thrombotic microangiopathy in severe aplastic anemia
Allergy & Immunology Cohort
Pretreatment sTNFR1 and infection predict transplant-associated thrombotic microangiopathy in severe aplastic anemia A simple blood test may flag a deadly transplant complication before it starts
This prospective nested case–control study evaluated patients with severe aplastic anemia undergoing allogeneic hematopoietic stem cell tran…
A blood marker measured before transplant may flag a deadly complication in severe aplastic anemia patients.
Frontiers Apr 27, 2026
Eltrombopag monotherapy assessed in moderate aplastic anemia and unilineage bone marrow failure disorders
Hematology Phase II
Eltrombopag monotherapy assessed in moderate aplastic anemia and unilineage bone marrow failure disorders Oral Drug May Reduce Need for Frequent Blood Transfusions
This Phase 2 trial evaluated eltrombopag by mouth once a day in 34 people at least 2 years of age with moderate aplastic anemia or unilineag…
A new oral medication offers hope for patients who rely on regular blood transfusions.
CT.gov Apr 24, 2026

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Questions about severe aplastic anemia

What clonal evolution patterns are seen in severe aplastic anemia treated with eltrombopag and immunosuppression?

Treatment with eltrombopag and immunosuppression leads to an increase in clonal mutations over time, with two main patterns of evolution: early changes involving chromosome 7 and late changes driven by pre-existing clones that can progress to cancer or PNH.

Full answer →