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

Background: 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).

Methods: 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).

Results: 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.

Conclusions: Dual haplo-HCT and living donor liver transplantation (LDLT) is feasible in eligible SCD patients with end-stage liver disease.