Has stem cell transplantation come of age in the treatment of sickle cell disease?

Shenoy, Shalini

doi:10.1038/sj.bmt.1705779

Cited by 30 publications

(23 citation statements)

References 61 publications

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“…As an example, strategies such as immunoablative regimens with alemtuzumab that can partially deplete immunoreactive graft T cells or posttransplantation cyclophosphamide as GVHD prophylaxis are under evaluation. 33,34 Mobilized peripheral blood products have a higher risk of chronic GVHD and mortality, especially in children. For this reason, pediatric transplantation protocols that use unmanipulated stem cell products have preferred to use BM, especially in nonmalignant disorders whenever feasible.…”

Section: Scope Of the Disease In Relation To Transplantation And Timingmentioning

confidence: 99%

Hematopoietic Stem Cell Transplantation for Sickle Cell Disease: Current Practice and Emerging Trends

Shenoy

2011

Hematology

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Allogeneic HSCT controls sickle cell disease (SCD)-related organ damage and is currently the only curative therapy available. Over the last 2 decades, HSCT has been limited largely to myeloablative matched sibling donor (MSD) procedures that are feasible only in a minority of patients. As the natural history of the disease has evolved, it is clear that subsets of patients with severe disease are at risk for sudden death, devastating CNS and pulmonary complications, and debilitating vasoocclusive crises. For these patients, the benefits of transplantation can outweigh the risks if HSCT can be safely and successfully performed with low early and late toxicities. This review describes advances and ongoing investigation of HSCT for SCD from the perspectives of recipient age and presentation, donor stem cell source, intensity of conditioning, family and medical perspectives, and other variables that influence outcome. Ultimately, HSCT should be viewed as a viable treatment option for SCD on par with other therapies for select patients who can benefit from the procedure.

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Section: Scope Of the Disease In Relation To Transplantation And Timingmentioning

confidence: 99%

Hematopoietic Stem Cell Transplantation for Sickle Cell Disease: Current Practice and Emerging Trends

Shenoy

2011

Hematology

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“…In comparison, 3-year posttransplant OS in adults with SCD is only 65%, suggesting that HSCT outcomes decrease as patients age. 25 This is likely due to the accumulated morbidity from SCD over time, which significantly limits tolerability and subsequent success of HSCT in adulthood. 26 Higher survival can be achieved with reduced-intensity regimens, but longterm success decreases and time on immune suppresion increases.…”

Section: Introductionmentioning

confidence: 99%

Lessons Learned From Talking With Parents About the Role of Hematopoietic Stem Cell Transplantation in the Treatment of Children With Sickle Cell Disease

Friedrich

Steinfield

Kim

et al. 2015

American Journal of Health Education

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Background: Hematopoietic stem cell transplantation (HSCT) is currently the only cure for sickle cell disease (SCD), but only a fraction of eligible children proceed to transplantation. We aimed to understand parental awareness and perceptions as a contributor. Purpose: To discuss HSCT with parents of children with SCD and assess their awareness and perceptions. Methods: A standardized educational session and pre-post surveys were used. Results: Fifty-seven parents completed the educational session. Parental awareness of HSCT and independent search for information were poor; only 40% of parents were previously aware of HSCT as a treatment option in SCD. However, 91% reported that the information was valuable, and 67% that they would consider HSCT as a treatment option for their child. Parents' main concerns were death, isolation, and infertility. Discussion: Our results support the use of structured educational materials during routine SCD health visits to provide information to parents about HSCT and begin discussing complex treatment options. Translation to Health Education Practice: Our results are relevant to clinicians, health educators, and patient advocates responsible for counseling parents of children with chronic conditions about high-risk curative options. A patient education sheet was generated from this experience and will be used to standardize ongoing parental education at our institutions.

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“…Reduced doses of busulfan (from 14 mg/kg to 6.4 mg/kg) appeared to allow engraftment, 29 whereas a similar reduction in melphalan dosing did not. 30 Since then, a bewildering number of conditioning regimens have been developed, mostly in the setting of malignant disease, in an attempt to bring transplantation procedures to a greater number of patients and potentially to those with SCD. To allow comparisons across studies, a recent categorization of conditioning regimens was proposed that used the terms myeloablative (MA), reduced-intensity conditioning, and nonmyeloablative (NMA).…”

Section: Conditioning Regimensmentioning

confidence: 99%

Allogeneic hematopoietic stem cell transplantation for sickle cell disease: the time is now

Hsieh

Fitzhugh

Tisdale

2011

Blood

128

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Although sickle cell disease (SCD) has a variable clinical course, many patients develop end-organ complications that are associated with significant morbidity and early mortality. Myeloablative allogeneic HSCT (allo-HSCT) is curative but has been historically performed only in children younger than 16 years of age. Modest modifications in the conditioning regimen and supportive care have improved outcome such that the majority of children with a suitable HLA-matched sibling donor can expect a cure from this approach. However, adult patients have been excluded from myeloablative allo-HSCT because of anticipated excess toxicity resulting from accumulated disease burden. Efforts to use nonmyeloablative transplantation strategies in adults logically followed but were initially met with largely disappointing results. Recent results, however, indicate that nonmyeloablative allo-HSCT in adult patients with SCD allows for stable mixed hematopoietic chimerism with associated full-donor erythroid engraftment and normalization of blood counts, and persistence in some without continued immunosuppression suggests immunologic tolerance. The attainment of tolerance should allow extension of these potentially curative approaches to alternative donor sources. Efforts to build on these experiences should increase the use of allo-HSCT in patients with SCD while minimizing morbidity and mortality. (Blood. 2011;118(5): 1197-1207)

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Has stem cell transplantation come of age in the treatment of sickle cell disease?

Cited by 30 publications

References 61 publications

Hematopoietic Stem Cell Transplantation for Sickle Cell Disease: Current Practice and Emerging Trends

Hematopoietic Stem Cell Transplantation for Sickle Cell Disease: Current Practice and Emerging Trends

Lessons Learned From Talking With Parents About the Role of Hematopoietic Stem Cell Transplantation in the Treatment of Children With Sickle Cell Disease

Allogeneic hematopoietic stem cell transplantation for sickle cell disease: the time is now

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