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Haplo BMT as first treatment for severe aplastic anemia

New clinical study proposes a shift in severe aplastic anemia treatment

A recent clinical study has the potential to change how doctors treat severe aplastic anemia (SAA)—and expand access to more patients.

SAA is a serious blood disorder in which a person’s bone marrow does not make enough new blood cells for the body. This can happen when the immune system attacks and destroys healthy blood stem cells inside the bone marrow. If left untreated, SAA can be fatal, with a death rate of approximately 70% within two years.

Historically, doctors used immunosuppressive therapy (IST) as the first treatment for SAA.  This treatment can initially be effective for many patients, but its long-term effectiveness is less than ideal. If IST is unsuccessful, the alternative has often been a bone marrow transplant (BMT) using a human leukocyte antigen (HLA)-matched donor.

BMT has significant potential in treating SAA, especially for younger patients. Yet, due to possible complications, it has typically been considered a secondary treatment option. Some patients also don’t have an HLA-matched donor—especially those who are racially and ethnically diverse—so BMT was not an option.

A clinical study’s results proposes a change in this approach.

Study overview

Duration: The study was conducted at one center in the United States between August 2016 and July 2020.

Participants: A total of 27 patients participated. Twenty were part of the formal trial. An additional seven received treatment based on the trial's guidelines. More than 35% of patients had diverse ancestries.

Procedure: Rather than beginning with IST, these patients underwent BMT from haploidentical (also known as half-matched or haplo) related donors.

Findings: The results indicate that the direct-to-BMT approach may be superior to traditional methods.

Implications of the study

  1. Increased access to treatment: Using donors other than just HLA-matched donors will allow more patients to benefit from BMT.
  2. Revised treatment strategy: The positive outcomes suggest that the medical community should consider re-evaluating the conventional treatment order for SAA. BMT might not need to be the last resort.
  3. Potential for improved outcomes: This approach might offer better results, especially for patients who are racially and ethnically diverse who may not always have immediate access to perfectly matched donors.

While further research is needed, this study represents a potentially significant shift in how doctors treat SAA. It emphasizes the potential benefits of reconsidering BMT as a primary treatment option, rather than a secondary one.

Read the clinical summary

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DeZern AE, Zahurak M, Symons HJ, et al. Alternative donor BMT with posttransplant cyclophosphamide as initial therapy for acquired severe aplastic anemia. Blood. 2023;141(25):3031-3038. doi: 10.1182/blood.2023020435.