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Haplo-stem cell transplant post liver transplantation to cure sickle cell disease with related liver dysfunction: a case series

Year: 2025

Type: article

Abstract: Sickle cell disease (SCD) is debilitating, with age-dependent complications such as stroke and liver disease, leading to significant morbidity and early mortality in young adults. Hematopoietic stem cell transplantation (HCT) is curative treatment for SCD, but transplantation-related risks often deter its use, especially in patients with severe comorbidities. A subset of severe SCD patients with significant end-organ dysfunction may benefit from a combined approach of HCT and solid organ transplantation (SOT). To the best of our knowledge, this report presents, for the first time, the utilization of dual orthotopic liver transplantation and haploidentical HCT (haplo-HCT) for severe SCD with advanced liver cirrhosis. Employing nonmyeloablative conditioning regimen and post-transplantation cyclophosphamide (PTCy). Both patients undergo orthotopic liver transplantation followed by haplo-HCT from the same related donors, achieve stable allograft function, full donor engraftment, and successful immunosuppression withdrawal through immune tolerance induction. Dual haplo-HCT and living donor liver transplantation (LDLT) is feasible in eligible SCD patients with end-stage liver disease. Sickle cell disease (SCD) affects the red blood cells resulting in pain and increased risk of infection. Transplant with bone marrow stem cells can cure SCD. Liver transplantation is also beneficial for people with liver failure. We combined transplant of liver and stem cells from the same donor in people with severe SCD and advanced liver disease. People benefited from this treatment, demonstrating this approach could be used for other people with SCD and liver disease. Alahmari et al. combine orthotopic liver transplantation with haploidentical hematopoietic stem cell transplant in patients with severe SCD and advanced liver disease. They demonstrate the feasibility of this dual approach, with stable engraftment and immune tolerance achieved through a nonmyeloablative conditioning regimen.

Source: Communications Medicine

DOI: 10.1038/s43856-025-01040-0