How I treat sickle cell disease with hematopoietic cell transplantation

Abstract

Sickle cell disease (SCD) leads to significant morbidity and early mortality, and hematopoietic cell transplantation (HCT) is the only widely available cure, with impacts seen on SCD-related organ dysfunction. Outcomes are excellent following matched-related donor (MRD) HCT, leading to significantly expanded application of this treatment over the past decade. The majority of SCD patients lack an MRD, but outcomes following alternative donor HCT continue to improve on clinical trials. Within this framework, we aim to provide our perspective on how to apply research findings to clinical practice, for an individual patient. We also emphasize that the preparation of SCD recipients for HCT and supporting them through HCT have special nuances that require awareness and close attention. Through the use of clinical vignettes, we provide our perpsective on the complex decision-making process in HCT for SCD as well as recommendations for the evaluation and support of these patients through HCT.

Graphical abstract

Figure 1.

Dramatic increase in number of SCD patients undergoing HCT. In data courtesy of the Center for International Blood and Marrow Transplantation, the annual number of transplants for SCD has quadrupled in the last decade of available data. The data presented here are preliminary and were obtained from the Coordinating Center of the Center for International Blood and Marrow Transplant Research. The analysis has not been reviewed or approved by the Statistical or Scientific Committees of the CIBMTR. No., number.

Figure 2.

Algorithm for consideration of HCT for SCD. Consideration of HCT and other curative therapies in individuals with SCD must take into consideration severity of disease to date, available HCT donor options, and clinical trials that an individual patient may be eligible for. While this decision-making process is complex, this algorithm provides a framework for how we consider an individual patient. Additionally, this algorithm does not take into account multiple processes that may occur concurrently, such as searching for URD and haploidentical donors, as well as considering gene therapy and mismatched related or unrelated donor HCT on a clinical trial.