Rapid early hematological response in a newly diagnosed patient with very severe aplastic anemia: a case report of high-dose romiplostim, hetrombopag and IST combination therapy - Report - MDSpire

Rapid early hematological response in a newly diagnosed patient with very severe aplastic anemia: a case report of high-dose romiplostim, hetrombopag and IST combination therapy

  • By

  • Liangkui Luo

  • Kaikai Huang

  • Yanbin Pang

  • May 25, 2026

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Clinical Report: Prompt Hematological Improvement in Very Severe Aplastic Anemia

Overview

This case study presents a 16-year-old male with very severe aplastic anemia (VSAA) who achieved rapid hematological improvement following treatment with high-dose romiplostim, hetrombopag, and immunosuppressive therapy. The patient reached complete remission by week 22, demonstrating the potential efficacy of this combination therapy in HSCT-ineligible patients.

Background

Very severe aplastic anemia (VSAA) is a critical bone marrow failure syndrome with limited treatment options for patients who are not candidates for allogeneic hematopoietic stem cell transplantation (HSCT). The standard immunosuppressive therapy (IST) often yields poor outcomes, necessitating the exploration of alternative therapeutic strategies. This case highlights the urgent need for effective treatments that can induce rapid responses in VSAA patients.

Data Highlights

Time PointNeutrophils (×10^9/L)Hemoglobin (g/L)Platelets (×10^9/L)
Week 00.138411
Week 41.40--
Week 9-7120
Week 12-7120
Week 181.00111117
Week 22-127160

Key Findings

  • A 16-year-old male with newly diagnosed VSAA achieved complete remission by week 22 after treatment.
  • The treatment regimen included high-dose romiplostim, hetrombopag, and standard immunosuppressive therapy.
  • Transfusion independence for red cells and platelets was achieved by week 12.
  • Throughout treatment, the patient experienced only mild side effects (grade 1–2 nausea and muscle spasms).
  • This case suggests that combining TPO-RAs may enhance hematologic recovery in VSAA patients.

Clinical Implications

The findings from this case report indicate that the combination of high-dose romiplostim and hetrombopag with IST may provide a viable treatment option for patients with VSAA who are ineligible for HSCT. Clinicians should consider this approach for rapid hematological improvement in similar patient populations.

Conclusion

This case underscores the potential of a dual TPO-RA regimen in achieving rapid responses in VSAA patients, warranting further investigation as a first-line treatment option for those unable to undergo HSCT.

Related Resources & Content

  1. American Society of Hematology 2026 Guidelines for the Diagnosis and Management of Severe Acquired Aplastic Anemia - PubMed
  2. the asco post — Romiplostim May Improve Chemotherapy-Induced Thrombocytopenia in Patients With GI Cancers
  3. The ASCO Post — Romiplostim May Improve Chemotherapy-Induced Thrombocytopenia in Patients With GI Cancers
  4. The ASCO Post — Romiplostim May Improve Chemotherapy-Induced Thrombocytopenia in Patients With GI Cancers
  5. The ASCO Post — Romiplostim May Improve Chemotherapy-Induced Thrombocytopenia in Patients With GI Cancers
  6. Romiplostim May Improve Chemotherapy-Induced Thrombocytopenia in Patients With GI Cancers
  7. American Society of Hematology 2026 Guidelines for the Diagnosis and Management of Severe Acquired Aplastic Anemia - PubMed
  8. Efficacy and safety of immunosuppressive therapy versus cyclosporine combined with avatrombopag in older adults with severe aplastic anemia: a multicenter prospective study | Blood Cancer Journal

Original Source(s)

Rapid early hematological response in a newly diagnosed patient with very severe aplastic anemia: a case report of high-dose romiplostim, hetrombopag and IST combination therapy

  • by Liangkui Luo, Kaikai Huang, Yanbin Pang

  • May 25, 2026

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