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Recent Research

Stay up-to-date on the advancing field of HCT with short summaries and links to the most relevant published research.

  • Allogeneic HCT should be considered as first-line treatment for SAA

    September 2018

    Researchers 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

    Read More

  • Consensus Statement on HCT Late Effects Screening Guidelines in Hemoglobinopathies

    June 2018
    Shenoy 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.

    Read More

  • HCT Can Be an Effective Treatment for Young Adults with Immune Deficiencies

    April 2018
    Fox 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.

    Read More

  • HCT Significantly Improves Quality of Life in Children with Sickle Cell Disease

    December 2017
    Bernaudin 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.

    Read More

  • Prognostic Factors Identified for Optimal HCT Outcomes in Adults with FA

    December 2017
    Bierings 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.

    Read More

  • 90% Survival, Low GVHD at Two Years after Alternative Donor HCT for SCD

    August 2017
    Gilman 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%.

    Read More

  • Case Study Review: How I Treat Severe Aplastic Anemia

    February 2017
    Bacigalupo 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).

    Read More

  • Large-Scale Study Shows Greater Than 90% Survival After HCT for Sickle Cell Disease

    January 2017
    Gluckman 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.”

    Read More

  • Alternative Donor HCT Better than Immunosuppressive Therapy in Pediatric SAA

    November 2016
    Choi 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).

    Read More

  • One-Year OS of 86% Achieved with Unrelated Donor HCT for Children with SCD

    October 2016
    Shenoy 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.

    Read More

  • HCT Outcomes in Older Patients with SAA Comparable to Younger Patients

    July 2016
    Shin 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).

    Read More

  • Expanding Use of HCT to Treat Sickle Cell Disease

    June 2016
    Arnold 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%.

    Read More

  • Improved Quality of Life, No GVHD after HCT in Adults with Sickle Cell Disease

    March 2016
    Saraf 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.

    Read More

  • Three-Year Survival of 94% in HCT for Sickle Cell Disease

    December 2015
    Cappelli 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%.

    Read More

  • Early HCT for SCD in Children is Preferred Therapy Prior to Onset of SCD Complications

    December 2015
    Satwani 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.

    Read More

  • 95% One-Year HCT Survival for Adults with Sickle Cell Disease

    December 2015
    Krishnamurti 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.

    Read More

  • Review: HLA-Identical Sibling HCT to Treat Sickle Cell Disease in Children

    November 2015
    Walters 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.

    Read More

  • Improved Outcomes in Alternative Donor HCT for Fanconi Anemia

    July 2015
    MacMillan 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).

    Read More

  • Excellent Survival in Pediatric Cord Blood Transplant for Non-Malignant Disorders

    April 2015
    Martinez 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.

    Read More

  • Excellent HCT Outcomes in Fanconi Anemia without Radiation

    April 2015
    Bonfim 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.

    Read More

  • Long-Term Study of HCT in Hurler Syndrome: Impact of Timing and Non-Carrier Donors

    April 2015
    Aldenhoven 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).

    Read More

  • Treosulfan-Based Conditioning and HCT for Nonmalignant Diseases

    December 2014
    Burroughs 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.

    Read More

  • Early Transplant Best in SCID Infants

    August 2014
    Pai 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.

    Read More

  • Novel Conditioning Regimen Permits HCT for Adults with Severe SCD

    July 2014
    Hsieh 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.

    Read More

  • HCT in Sickle Cell Disease and Thalassemia: Evidence-Based Focused Review

    May 2014
    King 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.

    Read More

  • Early Transplant in SCID Infants Free from Infections Results in Excellent Outcomes

    March 2014
    Pai 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.

    Read More

  • Longer-Term HCT Outcomes for Severe Sickle Cell Disease in Childhood

    February 2014
    Dedeken 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.

    Read More

  • Reduced-Intensity HCT for Chronic Granulomatous Disease

    February 2014
    Güngör 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.

    Read More

  • Review: Options for Patients with Refractory Aplastic Anemia

    December 2013
    Marsh 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.

    Read More

  • Comparable Outcomes for Unrelated, Sibling HCT in Aplastic Anemia

    December 2013
    Bacigalupo 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.

    Read More

  • Report: The Role of HCT in Treating HIV

    November 2013
    Zou 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.

    Read More

  • Understanding Factors in Long-Term HCT Outcome in Fanconi Anemia

    November 2013
    Peffault 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.

    Read More

  • Review: How I Treat SCID

    October 2013
    Gaspar 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).

    Read More

  • QOL More than 20 Years After HCT for Thalassemia

    September 2013

    La 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.

    Read More

  • New Autoimmune Diseases after Cord Blood Transplantation

    January 2013
    Daikeler 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.

    Read More

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Choose a Topic

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