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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Allogeneic HCT should be considered as first-line treatment for SAA
September 2018Researchers suggest the initial treatment guidelines for patients newly diagnosed with severe aplastic anemia (SAA) should be revised. They recommend allogeneic hematopoietic cell transplantation (HCT) as the first-line therapy and HLA typing for all newly diagnosed patients prior to starting treatment. (Georges GE, et al. Blood Advances)
Learn why the researchers are recommending the new first-line treatment strategy
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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. -
HCT Can Be an Effective Treatment for Young Adults with Immune Deficiencies
April 2018Fox TA, et al. Blood – Young adults with primary immunodeficiencies (PIDs) undergoing allogeneic hematopoietic cell transplantation (HCT) can achieve a 3-year overall survival greater than 80%, according to a study of 29 consecutive young adults transplanted at two U.K. hospitals. -
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. -
Prognostic Factors Identified for Optimal HCT Outcomes in Adults with FA
December 2017Bierings M, et al. Brit J Haematol – In this multicenter, retrospective study of adults with Fanconi anemia (FA) undergoing hematopoietic cell transplantation (HCT) between 1991 and 2014, researchers found that best outcomes were in patients who received bone marrow from an identical sibling with fludarabine in the conditioning regimen and transplanted after the year 2000. -
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%. -
Case Study Review: How I Treat Severe Aplastic Anemia
February 2017Bacigalupo A. Blood – In this installment of the “How I treat …” series in Blood, the author outlines his treatment algorithm for patients with severe aplastic anemia, using 2 case studies to illustrate the benefits and limitations of immunnosupressive therapy (IST) and hematopoietic cell transplantation (HCT). -
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.” -
Alternative Donor HCT Better than Immunosuppressive Therapy in Pediatric SAA
November 2016Choi YB, et al. Bone Marrow Transplant – This retrospective single-center study of children and adolescents with severe aplastic anemia (SAA) found that upfront hematopoietic cell transplantation (HCT) using alternative donors resulted in significantly better outcomes compared to both salvage HCT and upfront immunosuppressive treatment (IST). -
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. -
HCT Outcomes in Older Patients with SAA Comparable to Younger Patients
July 2016Shin SH, et al. Bone Marrow Transplant – A study of 117 adults with severe aplastic anemia (SAA) undergoing lower-intensity HCT using HLA-matched sibling donors found that transplant outcomes were comparable between older patients (>40 years) and younger patients (≤40 years). -
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. -
95% One-Year HCT Survival for Adults with Sickle Cell Disease
December 2015Krishnamurti L, et al. ASH abstract oral presentation, December 2015 – One-year overall and event-free survival probabilities were both 95% after hematopoietic cell transplantation (HCT) in adults with sickle cell disease (SCD), according to results of a multi-center study reported 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. -
Improved Outcomes in Alternative Donor HCT for Fanconi Anemia
July 2015MacMillan ML, et al. Blood — This prospective study of 130 patients (age range 1-48) transplanted for Fanconi anemia (FA) has shown that over a period of 10 years, transplant toxicity has decreased and engraftment has improved, resulting in a 5-year overall survival (OS) of 58% (95% CI, 49-59). -
Excellent Survival in Pediatric Cord Blood Transplant for Non-Malignant Disorders
April 2015Martinez C, et al. Oral presentation, BMT Tandem Meetings — Pediatric patients with non-malignant disorders can achieve “excellent overall survival” of 96%, without significant GVHD, after cord blood transplantation, according to results of a prospective trial presented at the BMT Tandem Meetings. -
Excellent HCT Outcomes in Fanconi Anemia without Radiation
April 2015Bonfim C, et al. Oral presentation, BMT Tandem Meetings — Five-year 5-year overall survival was 87% in a single-center study of 103 consecutive patients with Fanconi anemia who underwent allogeneic bone marrow transplantation between 2003 and 2014 using chemotherapy-only conditioning. -
Long-Term Study of HCT in Hurler Syndrome: Impact of Timing and Non-Carrier Donors
April 2015Aldenhoven M, et al. Blood — In this multi-center study of patients with mucopolysaccharidosis type I-Hurler syndrome (MPS-IH), 217 patients were transplanted at a median age of 16 months (range, 2-47 months) with a median age at last follow-up of 9.2 years (range, 3-23 years). -
Treosulfan-Based Conditioning and HCT for Nonmalignant Diseases
December 2014Burroughs LM, et al. Biol Blood Marrow Transplant — Patients with nonmalignant diseases undergoing hematopoietic cell transplantation using treosulfan-based conditioning can achieve a 2-year survival of 90%, according to a prospective multi-center trial of 31 transplants. -
Early Transplant Best in SCID Infants
August 2014Pai S-Y, et al. N Engl J Med — This multi-center, retrospective study of 240 infants with severe combined immunodeficiency (SCID) has demonstrated a five-year transplant survival rate of 74% in all patients, with higher rates of 82-94% in select patient groups. -
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. -
Early Transplant in SCID Infants Free from Infections Results in Excellent Outcomes
March 2014Pai S-Y, et al. BMT Tandem Meetings abstract oral presentation, February 2014 - A multi-center retrospective study of 240 infants with severe combined immunodeficiency (SCID) has demonstrated that five-year transplant survival is 74% overall, but can reach 88-100% in select patient groups. -
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. -
Reduced-Intensity HCT for Chronic Granulomatous Disease
February 2014Güngör T, et al. Lancet -- Reduced-intensity allogeneic HCT for chronic granulomatous disease can lead to two-year survival rates exceeding 95%, according to the results of a prospective multi-center trial. -
Review: Options for Patients with Refractory Aplastic Anemia
December 2013Marsh JCW et al. Blood - This review discusses the therapeutic options for aplastic anemia patients not responding to first-line immunosuppressive therapy (IST) with antithymocyte globulin (ATG) and cyclosporin, and have severe cytopenias at six months after IST. -
Comparable Outcomes for Unrelated, Sibling HCT in Aplastic Anemia
December 2013Bacigalupo A, et al. ASH abstract oral presentation, December 2013 – Unrelated donor hematopoietic cell transplantation (HCT) and sibling donor HCT yield comparable outcomes in patients with acquired severe aplastic anemia, according to a study of 1,500 patients. -
Report: The Role of HCT in Treating HIV
November 2013Zou S, et al. Blood - This report contains the recommendations of a working group of the National Heart, Lung, and Blood Institute (NHLBI) convened to address emerging cellular therapies to treat HIV infection. -
Understanding Factors in Long-Term HCT Outcome in Fanconi Anemia
November 2013Peffault de Latour RP, et al. Blood - The estimated 20-year survival of 795 Fanconi anemia (FA) patients who underwent first HCT between 1972 and 2010 is 49%, according to this multi-center study published in Blood. -
Review: How I Treat SCID
October 2013Gaspar HB, et al. Blood - In this installment of the “How I Treat” series in the journal Blood, Dr. H. Bobby Gaspar and colleagues describe their treatment algorithms for pediatric patients with severe combined immunodeficiency (SCID). -
QOL More than 20 Years After HCT for Thalassemia
September 2013La Nasa G, et al. Blood - The long-term health-related quality of life (QOL) of beta-thalassemia patients who underwent allogeneic transplantation is similar to that of the general population, according to a study of 109 patients transplanted in the 1980s and 1990s.
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New Autoimmune Diseases after Cord Blood Transplantation
January 2013Daikeler T, et al. Blood - An analysis of 726 cord blood transplants reported to the European cord blood transplant database has found that the cumulative incidence of autoimmune disease developing post-transplant in these patients was 5.0% at 1 year and 6.6% at 5 years.