scholarly journals Hematopoietic cell transplantation for sickle cell disease: updates and future directions

Hematology ◽  
2021 ◽  
Vol 2021 (1) ◽  
pp. 181-189
Author(s):  
Lakshmanan Krishnamurti
Keyword(s):  
Sickle Cell Disease ◽  
Cell Transplantation ◽  
Sickle Cell ◽  
Hematopoietic Cell Transplantation ◽  
Ex Vivo ◽  
Autologous Transplantation ◽  
Hematopoietic Cell ◽  
Cell Disease ◽  
The Impact

Abstract Excellent outcomes in hematopoietic cell transplantation (HCT) from HLA-identical siblings, improvements in conditioning regimens, novel graft-versus-host disease prophylaxis, and the availability of alternative donors have all contributed to the increased applicability and acceptability of HCT for sickle cell disease (SCD). In young children with symptomatic SCD with an available HLA-identical related donor, HCT should be carefully considered. HCT from alternative donors is typically undertaken only in patients with severe symptoms, causing or likely to cause organ damage, and in the context of clinical trials. Patients undergoing HCT for SCD require careful counseling and preparation. They require careful monitoring of unique organ toxicities and complications during HCT. Patients must be prospectively followed for a prolonged time to determine the long-term outcomes and late effects of HCT for SCD. Thus, there is a need for a universal, longitudinal clinical registry to follow patients after HCT for SCD in conjunction with individuals who do not receive HCT to compare outcomes. Antibody-based conditioning and ex-vivo umbilical cord blood expansion are likely to improve the availability and acceptability of HCT. In addition, new disease-modifying drugs and the emerging option of the autologous transplantation of gene-modified hematopoietic progenitor cells are likely to expand the available therapeutic options and make decision-making by patients, physicians, and caregivers even more complicated. Future efforts must also focus on determining the impact of socioeconomic status on access to and outcomes of HCT and the long-term impact of HCT on patients, families, and society.

scholarly journals Stable Long-Term Donor Engraftment following Reduced-Intensity Hematopoietic Cell Transplantation for Sickle Cell Disease

2008 ◽  
Vol 14 (11) ◽  
pp. 1270-1278 ◽  
Author(s):  
Lakshmanan Krishnamurti ◽  
Sandhya Kharbanda ◽  
Melinda A. Biernacki ◽  
Wandi Zhang ◽  
K. Scott Baker ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Cell Transplantation ◽  
Sickle Cell ◽  
Hematopoietic Cell Transplantation ◽  
Hematopoietic Cell ◽  
Cell Disease ◽  
Reduced Intensity

Early Identification of Barriers and Facilitators to Self-Management Behaviors in Pediatric Patients With Sickle Cell Disease to Minimize Hematopoietic Cell Transplantation Complications

2018 ◽  
Vol 35 (3) ◽  
pp. 199-209 ◽  
Author(s):  
Ginny L. Schulz ◽  
Rebecca H. Foster ◽  
Valerie Kennedy Lang ◽  
Alison Towerman ◽  
Shalini Shenoy ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Cell Transplantation ◽  
Sickle Cell ◽  
Pediatric Patients ◽  
Hematopoietic Cell Transplantation ◽  
Hematopoietic Cell ◽  
Barriers And Facilitators ◽  
Self Management ◽  
Cell Disease ◽  
Management Behaviors

Hematopoietic cell transplantation (HCT) is an elective, curative treatment option for patients with sickle cell disease (SCD). Transplant requires extensive self-management behaviors to be successful. The purpose of this study was to describe potential barriers and facilitators to self-management in a group of pediatric patients with SCD prior to HCT and their medical outcomes post-HCT. A multiple case study approach was used to describe 4 pairs of transplant recipients grouped by age, donor type, and donor source. Each pair included a case with minimal and increased post-HCT complications. Complications included readmissions, graft-versus-host disease, systemic infections, and survival in the first year post-HCT. Variables were retrospectively collected and content analyzed to identify barriers and facilitators within and across pairs using existing self-management frameworks. While higher risk transplants experienced more complications, 3 of the 4 cases with increased complications had a larger number of modifiable barriers identified compared with those experiencing minimal complications. At least one modifiable barrier and multiple facilitators were identified in all cases. A standardized psychosocial assessment process with an established plan to mitigate barriers and promote facilitators to self-management is essential to optimize outcomes in patients with SCD undergoing elective HCT.

scholarly journals Hematopoietic Cell Transplantation for Sickle Cell Disease

2021 ◽  
Vol 8 ◽  
Author(s):  
Lakshmanan Krishnamurti
Keyword(s):  
Sickle Cell Disease ◽  
Progenitor Cells ◽  
Cell Transplantation ◽  
Sickle Cell ◽  
Hematopoietic Cell Transplantation ◽  
Hematopoietic Cell ◽  
Hematopoietic Progenitor Cells ◽  
Cell Disease ◽  
Hematopoietic Progenitor ◽  
Erythrocyte Deformation

Sickle cell disease (SCD) is a severe autosomal recessively inherited disorder of the red blood cell characterized by erythrocyte deformation caused by the polymerization of the abnormal hemoglobin, which leads to erythrocyte deformation and triggers downstream pathological changes. These include abnormal rheology, vaso-occlusion, ischemic tissue damage, and hemolysis-associated endothelial dysfunction. These acute and chronic physiologic disturbances contribute to morbidity, organ dysfunction, and diminished survival. Hematopoietic cell transplantation (HCT) from HLA-matched or unrelated donors or haploidentical related donors or genetically modified autologous hematopoietic progenitor cells is performed with the intent of cure or long-term amelioration of disease manifestations. Excellent outcomes have been observed following HLA-identical matched related donor HCT. The majority of SCD patients do not have an available HLA-identical sibling donor. Increasingly, however, they have the option of undergoing HCT from unrelated HLA matched or related haploidentical donors. The preliminary results of transplantation of autologous hematopoietic progenitor cells genetically modified by adding a non-sickling gene or by genomic editing to increase expression of fetal hemoglobin are encouraging. These approaches are being evaluated in early-phase clinical trials. In performing HCT in patients with SCD, careful consideration must be given to patient and donor selection, conditioning and graft-vs.-host disease regimen, and pre-HCT evaluation and management during and after HCT. Sociodemographic factors may also impact awareness of and access to HCT. Further, there is a substantial decisional dilemma in HCT with complex tradeoffs between the possibility of amelioration of disease manifestations and early or late complications of HCT. The performance of HCT for SCD requires careful multidisciplinary collaboration and shared decision making between the physician and informed patients and caregivers.

scholarly journals Indications and Results of HLA-Identical Sibling Hematopoietic Cell Transplantation for Sickle Cell Disease

2016 ◽  
Vol 22 (2) ◽  
pp. 207-211 ◽  
Author(s):  
Mark C. Walters ◽  
Laura M. De Castro ◽  
Keith M. Sullivan ◽  
Lakshmanan Krishnamurti ◽  
Naynesh Kamani ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Cell Transplantation ◽  
Sickle Cell ◽  
Hematopoietic Cell Transplantation ◽  
Hematopoietic Cell ◽  
Cell Disease
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