Case Study: A Groundbreaking Approach Combining Haplo-HSCT and UC-MSCs Offers Hope for Severe Aplastic Anemia

Despite significant strides in immunosuppressive therapy, complete cures remain elusive for many SAA patients. Haplo-HSCT has emerged as a viable alternative, particularly for adult SAA patients. However, its efficacy is marred by a higher incidence of graft failure and graft-versus-host disease (GVHD). In response, researchers devised a novel treatment protocol, blending haplo-HSCT with UC-MSCs, and evaluated its impact on adult SAA patients.

The study enlisted 25 adult patients aged 18 and above diagnosed with SAA. They underwent haploidentical hematopoietic stem cell (HSC) transplantation coupled with UC-MSC infusion following a conditioning regimen comprising busulfan, cyclophosphamide, fludarabine, and anti-thymocyte globulin. Robust GVHD prophylaxis, including cyclosporine, basiliximab, mycophenolate mofetil, and short-term methotrexate, was also administered. The outcomes were then compared with those of juvenile SAA patients (N = 75).

Encouragingly, all patients achieved myeloid engraftment post haplo-HSCT, with a median engraftment time of 16.12 days (range, 11–26). Platelet engraftment followed suit at a median of 28.30 days (range, 13–143). The cumulative incidence of grade II acute GVHD (aGVHD) at day +100 stood at 32.00 ± 0.91%, with no instances of grade III–IV aGVHD recorded at the same juncture. Similarly, the cumulative incidence of total chronic GVHD was 28.00 ± 0.85%. Notably, the overall survival rate reached 71.78 ± 9.05% at a median follow-up of 42.08 months (range, 2.67–104). Impressively, the treatment protocol yielded comparable curative effects across both young and adult SAA patient cohorts.

In essence, the combination of haplo-HSCT and UC-MSC infusion emerges as a promising therapeutic strategy for SAA patients, offering renewed optimism in the quest for effective treatment modalities. With further research and refinement, this innovative approach holds the potential to transform outcomes and enhance the quality of life for individuals grappling with this challenging hematological condition.

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Source: https://www.sciencedirect.com/science/article/abs/pii/S1465324921008288

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