Haploidentical Hematopoietic Cell Transplantation for Sickle Cell Disease

2017 ◽  
pp. 283-302
Author(s):  
Elizabeth O. Stenger ◽  
Allistair Abraham
Keyword(s):  
Sickle Cell Disease ◽  
Cell Transplantation ◽  
Sickle Cell ◽  
Hematopoietic Cell Transplantation ◽  
Hematopoietic Cell ◽  
Cell Disease ◽  
Haploidentical Hematopoietic Cell Transplantation

scholarly journals Relationship between Mixed Donor–Recipient Chimerism and Disease Recurrence after Hematopoietic Cell Transplantation for Sickle Cell Disease

2017 ◽  
Vol 23 (12) ◽  
pp. 2178-2183 ◽  
Author(s):  
Allistair Abraham ◽  
Matthew Hsieh ◽  
Mary Eapen ◽  
Courtney Fitzhugh ◽  
Jeanette Carreras ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Cell Transplantation ◽  
Sickle Cell ◽  
Hematopoietic Cell Transplantation ◽  
Disease Recurrence ◽  
Hematopoietic Cell ◽  
Cell Disease

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 Primary caregiver decision‐making in hematopoietic cell transplantation and gene therapy for sickle cell disease

2020 ◽  
Vol 68 (1) ◽  
Author(s):  
Cynthia B. Sinha ◽  
Nitya Bakshi ◽  
Diana Ross ◽  
George Loewenstein ◽  
Lakshmanan Krishnamurti
Keyword(s):  
Decision Making ◽  
Gene Therapy ◽  
Sickle Cell Disease ◽  
Cell Transplantation ◽  
Sickle Cell ◽  
Hematopoietic Cell Transplantation ◽  
Primary Caregiver ◽  
Hematopoietic Cell ◽  
Cell Disease

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

scholarly journals Clinical risks and healthcare utilization of hematopoietic cell transplantation for sickle cell disease in the USA using merged databases

Haematologica ◽  
2017 ◽  
Vol 102 (11) ◽  
pp. 1823-1832 ◽  
Author(s):  
Staci D. Arnold ◽  
Ruta Brazauskas ◽  
Naya He ◽  
Yimei Li ◽  
Richard Aplenc ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Cell Transplantation ◽  
Healthcare Utilization ◽  
Sickle Cell ◽  
Hematopoietic Cell Transplantation ◽  
Hematopoietic Cell ◽  
Cell Disease ◽  
The Usa ◽  
Clinical Risks

Parent and Guardian Knowledge of Hematopoietic Cell Transplantation as a Treatment Option for Sickle Cell Disease

2019 ◽  
Vol 41 (3) ◽  
pp. 187-193 ◽  
Author(s):  
Alicia M. Stallings ◽  
Navneet S. Majhail ◽  
Amy S. Nowacki ◽  
Grace I. Onimoe ◽  
Rabi Hanna ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Cell Transplantation ◽  
Sickle Cell ◽  
Treatment Option ◽  
Hematopoietic Cell Transplantation ◽  
Hematopoietic Cell ◽  
Cell Disease
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