Unique Challenges of Hematopoietic Stem Cell Transplant for Sickle Cell Disease

2017 ◽  
pp. 185-198
Author(s):  
Pooja Khandelwal ◽  
Michael Grimley
Keyword(s):  
Stem Cell ◽  
Sickle Cell Disease ◽  
Hematopoietic Stem Cell ◽  
Sickle Cell ◽  
Hematopoietic Stem Cell Transplant ◽  
Stem Cell Transplant ◽  
Cell Transplant ◽  
Cell Disease ◽  
Hematopoietic Stem

Allogeneic hematopoietic stem cell transplant for sickle cell disease: The why, who, and what

Blood Reviews ◽  
2021 ◽  
pp. 100868
Author(s):  
Emanuela Cimpeanu ◽  
Maria Poplawska ◽  
Brian Campbell Jimenez ◽  
Dibyendu Dutta ◽  
Seah H. Lim
Keyword(s):  
Stem Cell ◽  
Sickle Cell Disease ◽  
Hematopoietic Stem Cell ◽  
Sickle Cell ◽  
Hematopoietic Stem Cell Transplant ◽  
Stem Cell Transplant ◽  
Cell Transplant ◽  
Cell Disease ◽  
Hematopoietic Stem

The ethics of a proposed study of hematopoietic stem cell transplant for children with “less severe” sickle cell disease

Blood ◽  
2014 ◽  
Vol 124 (6) ◽  
pp. 861-866 ◽  
Author(s):  
Robert S. Nickel ◽  
Jeanne E. Hendrickson ◽  
Ann E. Haight
Keyword(s):  
Stem Cell ◽  
Sickle Cell Disease ◽  
Hematopoietic Stem Cell ◽  
Sickle Cell ◽  
Hematopoietic Stem Cell Transplant ◽  
Stem Cell Transplant ◽  
Individual Autonomy ◽  
Cell Transplant ◽  
Cell Disease ◽  
Hematopoietic Stem

Abstract Hematopoietic stem cell transplant (HSCT) is the only cure for sickle cell disease (SCD). HSCT using an HLA-identical sibling donor is currently an acceptable treatment option for children with severe SCD, with expected HSCT survival >95% and event-free survival >85%. HSCT for children with less severe SCD (children who have not yet suffered overt disease complications or only had mild problems) is controversial. It is important to consider the ethical issues of a proposed study comparing HLA-identical sibling HSCT to best supportive care for children with less severe SCD. In evaluating the principles of nonmaleficence, respect for individual autonomy, and justice, we conclude that a study of HLA-identical sibling HSCT for all children with SCD, particularly hemoglobin SS and Sβ0-thalassemia disease, is ethically sound. Future work should explore the implementation of a large trial to help determine whether HSCT is a beneficial treatment of children with less severe SCD.

Improved Splenic Function After Hematopoietic Stem Cell Transplant for Sickle Cell Disease

2016 ◽  
Vol 63 (5) ◽  
pp. 908-913 ◽  
Author(s):  
Robert Sheppard Nickel ◽  
Elizabeth Seashore ◽  
Peter A. Lane ◽  
Adina L Alazraki ◽  
John T. Horan ◽  
...  
Keyword(s):  
Stem Cell ◽  
Sickle Cell Disease ◽  
Hematopoietic Stem Cell ◽  
Sickle Cell ◽  
Hematopoietic Stem Cell Transplant ◽  
Stem Cell Transplant ◽  
Cell Transplant ◽  
Cell Disease ◽  
Hematopoietic Stem ◽  
Splenic Function

scholarly journals The Role of Hematopoietic Stem Cell Transplantation in the treatment of Sickle Cell Disease

2020 ◽  
Vol 7 (09) ◽  
pp. 5024-5032
Author(s):  
Carolina Wishner ◽  
Colleen Taylor ◽  
Monica Williams ◽  
Derian Kuneman
Keyword(s):  
Stem Cell ◽  
Sickle Cell Disease ◽  
Hematopoietic Stem Cell ◽  
Sickle Cell ◽  
Hematopoietic Stem Cell Transplant ◽  
Organ Damage ◽  
Cell Transplant ◽  
Cell Disease ◽  
Hematopoietic Stem

Abstract   Sickle cell disease (SCD) affects millions of people around the world and is associated with significant morbidity and premature mortality. It is a chronic, life-long illness that affects virtually every tissue in the body, worsens over time, with varying degrees of morbidity in everyone with the disease.  Before hematopoietic stem cell transplant (HCST), the mainstay of the management of SCD included early identification of the disease through newborn screening, infection prophylaxis with vaccinations and antibiotics, management of pain crises, blood transfusions, and hydroxyurea.   These treatments although beneficial, do not cure SCD, stop the progressive end-organ damage associated with this disease, and are lifelong.   Hematopoietic stem cell transplant is one of two treatment options that offer a cure for SCD and stops the progressive end-organ damage. The purpose of this article is to examine traditional treatments (best medical practice) and HCST for SCD and their associated complications.  The role of HCST in the treatment of sickle cell disease, as well as recent research on HSCT as a cure for SCD, risk factors, patient selection, limitations, and future use of this treatment option, are also reviewed.  Major issues surrounding the use of HCST for treating SCD include the optimal age for transplantation, disease severity, donor source, and the conditioning regimen before transplantation. The future of HCST including gene therapy is also discussed.

Higher Prevalence of Hydroxyurea Use Is Associated with Lower Hospitalization Rate in a Population of Children with Sickle Cell Disease

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 315-315
Author(s):  
Cecelia Calhoun ◽  
Ryan Colvin ◽  
Arti Verlekar ◽  
Sherry Lassa-Claxton ◽  
F. Sessions Cole ◽  
...  
Keyword(s):  
Stem Cell ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Hematopoietic Stem Cell Transplant ◽  
Stem Cell Transplant ◽  
Hospitalization Rate ◽  
Cell Transplant ◽  
Cell Disease ◽  
Hematopoietic Stem ◽  
Genotype Group

Abstract Higher Prevalence of Hydroxyurea Use Is Associated with Lower Hospitalization Rate in a Population of Children with Sickle Cell Disease Cecelia L. Calhoun, MD; Ryan Colvin, MPH; Sherry Lassa-Claxton, MS; Arti Verlekar MS; F. Sessions Cole, MD; Monica L. Hulbert, MD Background Individuals with sickle cell disease (SCD) treated with hydroxyurea (HU) in clinical trials have had fewer hospitalizations and vaso-occlusive pain episodes than those on placebo. Since 2014, HU has been recommended for all children with Hemoglobin SS/S-β0 thalassemia, and many SCD centers have increased HU utilization. The effect of expanded HU treatment on hospitalization rate has not been reported in a population of children clinically treated with HU. In a pediatric SCD center cohort, we tested the hypothesis that increasing prevalence of HU treatment was associated with reduced hospitalization rate per patient-year and in total hospitalizations. Methods PedsNet, a PCORI-funded clinical research initiative, was queried for all patients with inpatient or outpatient encounters for SCD at St. Louis Children's Hospital (SLCH) from 2010 through 2015. Data from patients with ICD-9 codes indicating any genotype of SCD were included. Patients were considered to be receiving HU when it was prescribed on 2 or more outpatient encounters. Exclusion criteria were lack of SCD diagnosis confirmation, lack of outpatient visits at SLCH, and presence of a significant unrelated medical condition. Patients were censored at their 21st birthday or date of hematopoietic stem cell transplant. Data generated at the adult hospital were excluded. All hospitalizations in the final cohort were included for analysis, regardless of diagnosis code. For comparisons, patients were grouped as having a severe genotype (SS, S-β0 thalassemia, SD) or less-severe genotype (SC, S-β+ thalassemia). Statistical analysis was performed using SAS 9.4. Logistic and Poisson regression were used to compare proportions and rates between groups, respectively. Generalized Estimating Equations were used to account for within patient correlation. Results The PedsNet query identified 646 patients. Sixty patients were excluded due to lack of SCD diagnosis, 78 due to lack of outpatient care, and 4 due to severe unrelated disease (3 with congenital disease/malformation, 1 with cancer), leaving 504 who met all inclusion criteria (51.6% male). Hematopoietic stem cell transplant was performed in 23 patients during the study period. Of patients with a severe genotype, 28 (20.1%) received HU in 2010 compared with 111 (65.6%) in 2015 (p<0.0001). Fewer patients in the less-severe genotype group received HU: 5 patients (4.3%) in 2010 and 13 (14.3%) in 2015 (p=0.006). In the severe genotype group, the rate of hospitalizations per patient-year decreased from 1.71 to 1.01 (p<0.0001) and the total number of hospitalizations decreased from 345 to 197. Throughout the study period, HU-treated patients in the severe genotype group had a higher hospitalization rate than HU-untreated patients; both treated and untreated patients had a significantly lower rate in 2015 than 2010 (Table), suggesting that hospitalizations were prevented as children who were more prone to hospitalizations began HU therapy. There was no significant change in hospitalization rate over time in the less-severe genotype group, at 1.13 hospitalizations per patient-year in 2010 and 1.16 in 2015 (p=0.94), with no impact of HU in this group. Conclusions In this clinical cohort of children with SCD, increasing prevalence of HU treatment was associated with significantly fewer total hospitalizations and a lower hospitalization rate per patient-year. The constant hospitalization rate in the less-severe genotype group suggests that the reduction is attributable to HU, rather than to other changes in clinical practice affecting patients with all genotypes. Hospitalization rates were higher among HU-treated patients, suggesting that more symptomatic children remain more likely to receive HU despite published recommendations to treat all children with severe genotypes. Families and clinicians may be reluctant to initiate HU for primary prevention of pain and hospitalizations in asymptomatic children. Future work should examine HU effects on specific SCD complications and on costs of care. Family and clinician interventions are needed to promote HU utilization in targeted patient groups. Table. Table. Disclosures Hulbert: Pfizer, Inc.: Other: spouse employment.

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