Recent Research
Stay up-to-date on the advancing field of HCT with short summaries and links to the most relevant published research.
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Does age at time of HCT for patients with sickle cell disease impact outcomes?
June 2019Cappelli B, et al. Haematologica – The youngest of three age-based patient cohorts (age 0-5 years) in a study of 736 patients with sickle cell disease (SCD) undergoing HLA-identical sibling hematopoietic cell transplantation (HCT) experienced 4-year event-free survival of 93%, results the researchers characterized as “excellent.”
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Neurocognition stabilizes after HCT for patients for sickle cell disease-induced cognitive decline
February 2019In addition to the risk of stroke and cognitive decline, children with sickle cell disease (SCD) may lose approximately one IQ point per year on a full scale IQ (FSIQ) test. For the first time, researchers report stabilization of IQ and central nervous system (CNS) outcomes after unrelated allogeneic hematopoie
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How I Treat Older Adults with Sickle Cell Disease
October 2018Thein SL and Howard J., Blood Journal – In this installment of the “How I treat…” series, the authors highlight that for patients with sickle cell disease, survival has significantly improved. Consequently, adults face new long-term risks of their disease as well as age-related conditions.
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Consensus Statement on HCT Late Effects Screening Guidelines in Hemoglobinopathies
June 2018Shenoy S, et al. Biol Blood Marrow Transplant – An international panel of HCT experts has updated late effects screening guidelines for pediatric transplantation for hemoglobinopathies, focusing on sickle cell disease and thalassemia. -
Ethical Challenges in HCT for Sickle Cell Disease
February 2018Nickel RS, et al. Biol Blood Marrow Transplant – People with sickle cell disease (SCD) experience a shortened life expectancy, painful and debilitating symptoms, and a lowered quality of life. Thus, clinicians face ethical dilemmas when counseling their patients with SCD on whether to pursue HCT, which is currently the only potential cure for the disease, but which comes with a risk of mortality. -
Study Identifies Key Factors Influencing the Decision to Pursue HCT for Sickle Cell Disease
January 2018Khemani K, et al. Biol Blood Marrow Transplant – Currently, hematopoietic stem cell transplant (HCT) is the only treatment for sickle cell disease (SCD) with a curative intent. -
HCT Significantly Improves Quality of Life in Children with Sickle Cell Disease
December 2017Bernaudin F, et al. ASH abstract oral presentation, December 2017 – Children with sickle cell disease (SCD) have significantly better physical health and school functioning after transplantation than do those receiving chronic transfusion, according to results of a donor/no donor study of 67 patients with SCD. -
90% Survival, Low GVHD at Two Years after Alternative Donor HCT for SCD
August 2017Gilman AL, et al. Blood Advances – A study of patients with symptomatic sickle cell disease (SCD) who underwent CD34+ cell-selected, T-cell–depleted peripheral blood HCT from a mismatched family member or unrelated donor has resulted in a 2-year overall survival rate of 90%. -
Large-Scale Study Shows Greater Than 90% Survival After HCT for Sickle Cell Disease
January 2017Gluckman E, et al. Blood – Researchers analyzing the outcomes of 1,000 patients with sickle cell disease (SCD) who underwent identical sibling hematopoietic cell transplantation (HCT) between 1986 and 2013 characterized the 5-year survival as “excellent” and concluded that their results “confirm this is an accepted treatment for severe SCD.” -
One-Year OS of 86% Achieved with Unrelated Donor HCT for Children with SCD
October 2016Shenoy S, et al Blood – This multi-center phase II trial conducted by the Blood and Marrow Transplant Clinical Trials Network (BMT CTN) analyzed outcomes of 29 children (age range 6-19 years) with sickle cell disease (SCD) who underwent reduced-intensity conditioning unrelated donor hematopoietic cell transplantation (HCT) between 2008 and 2014. -
Expanding Use of HCT to Treat Sickle Cell Disease
June 2016Arnold SD, et al. Br J Haematol – In this review of hematopoietic cell transplantation (HCT) for sickle cell disease (SCD), the authors outline the indications for transplantation in both children and adults, and note that overall survival in matched sibling HCT exceeds 90%. -
Improved Quality of Life, No GVHD after HCT in Adults with Sickle Cell Disease
March 2016Saraf SL, et al. Biol Blood Marrow Transplant – HCT using an innovative conditioning regimen can result in a cure with marked improvements in quality of life for patients with high-risk sickle cell disease, according to a study of 13 adults transplanted using matched related donors. -
Three-Year Survival of 94% in HCT for Sickle Cell Disease
December 2015Cappelli B, et al. ASH abstract oral presentation, December 2015 – Results presented at ASH from a study of 1,000 matched sibling donor transplants in patients with severe sickle cell disease (SCD) have demonstrated a three-year overall survival of 94% and an event-free survival of 90%. -
Early HCT for SCD in Children is Preferred Therapy Prior to Onset of SCD Complications
December 2015Satwani P, et al. ASH abstract oral presentation, December 2015 – Hematopoietic cell transplantation (HCT) to treat sickle cell disease (SCD) can result in up to 96% survival at two years, according to research results presented at the ASH Annual Meeting. -
Review: HLA-Identical Sibling HCT to Treat Sickle Cell Disease in Children
November 2015Walters MC, et al. Biol Blood Marrow Transplant – In this of review of HLA-identical sibling transplantation to treat sickle cell disease (SCD) in children, the authors assess the risk of mortality and compare published rates of survival in SCD patients treated and not treated by HCT. -
Novel Conditioning Regimen Permits HCT for Adults with Severe SCD
July 2014Hsieh MM, et al. JAMA — In this prospective study of 30 patients with severe sickle cell disease (SCD), a novel non-myeloablative conditioning regimen prior to HCT was shown to reduce regimen-related toxicity and result in stable long-term donor mixed T-cell chimerism. -
HCT in Sickle Cell Disease and Thalassemia: Evidence-Based Focused Review
May 2014King A, et al. Blood — An evidence-based review of the status of HCT as treatment of sickle cell disease (SCD) and thalassemia presented in two case studies to introduce possible treatment algorithms based on peer-reviewed, published methods and outcomes of HCT for each disease. -
Longer-Term HCT Outcomes for Severe Sickle Cell Disease in Childhood
February 2014Dedeken L, et al. Br J Haematol -- Allogeneic HCT for sickle cell disease (SCD) can lead to survival rates exceeding 90%, according to results of a single-center study of 50 children with severe SCD.