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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Review: Scientific advances in myelodysplastic syndromes
March 2019Löwenberg B, Blood Journal – A series of review articles in the Blood Journal provides a comprehensive overview of the current landscape of MDS, highlighting advances in biological and clinical science.
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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 Philadelphia chromosome positive ALL
January 2019The introduction of effective novel agents is swiftly changing management of patients with Philadelphia chromosome positive acute lymphoblastic leukemia (Ph+ ALL). In this edition of the “How I treat…” series in the Blood Journal , the author uses case studies to address questions about Ph+ ALL treatment deci
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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. -
Second HCT Effective for Subset of Relapsing Children with Acute Leukemia
April 2018Yaniv I, et al. Biol Blood Marrow Transplant – This multi-center study of 373 children who relapsed after allogeneic hematopoietic cell transplantation (HCT) revealed several prognostic factors predicting overall survival and leukemia-free survival in a subset of these patients. -
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. -
Personalized HCT Prognostic Risk Scores Identified for Children with Acute Leukemias
January 2018Bitan M, et al. Biol Blood Marrow Transplant – Researchers analyzed outcomes of children with acute myeloid (AML, n=790) and acute lymphoblastic leukemia (ALL, n=1,096) who survived for at least 1 year in remission after hematopoietic cell transplantation (HCT) have developed personalized long-term prognostic scoring systems for these patients. -
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. -
Study Identifies Optimal Therapies for Relapsed Pediatric AML
September 2017Karlsson L, et al. Br J Haematol – A large-scale study of children with relapsed acute myeloid leukemia (AML) has found that intensive re-induction therapy followed by hematopoietic cell transplantation (HCT) in second complete remission (CR2) is the best available treatment for the majority of these patients. -
30% of Very Young HCT Recipients Experience One or More Late Effects
June 2017Vrooman LM, et al. Biol Blood Marrow Transplant - This multi-center study showed that children under 3 years old who undergo HCT are at high risk for late effects. After 1 year, 30% had developed 1 or more organ toxicity or transplant-related complication. -
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). -
Improved Survival Over Time in Pediatric HCT
July 2016Svenberg P, et al. Ped Transplant – A long-term study of pediatric allogeneic transplants found that outcomes improved over time, with significantly improved overall survival (OS) in transplants performed in the 2003-2013 time period compared to those performed in 1992-2002. -
Better HCT Outcomes Over Time in Children and Adolescents
March 2016Brissot E, et al. Bone Marrow Transplant – Major advances in supportive care and HCT practice in the past decade have resulted in expanded indications and a significant survival benefit for pediatric patients undergoing allogeneic HCT, according to a single-center study of 250 children and adolescents transplanted between 1983 and 2010. -
Low Incidence of Late Cardiovascular Complications after Pediatric HCT
February 2016Duncan CN, et al. BMT Tandem Meetings abstract oral presentation, February 2016 – Serious cardiovascular (CV) late effects are uncommon in pediatric transplant survivors, according to a multi-center study of 661 children who underwent hematopoietic cell transplantation (HCT) for hematologic malignancy between 1995 and 2008. -
Children Under 3 Years Old Undergoing HCT Require Long-Term Monitoring for Late Effects
February 2016Vrooman L, et al. BMT Tandem Meetings abstract oral presentation, February 2016 – A study of myeloablative hematopoietic cell transplantation (HCT) performed in children under 3 years old has found that although transplant-related mortality at 10 years is only 5%, recipients require monitoring for late complications. -
Children’s Oncology Group: Long-Term Follow-Up Guidelines After Pediatric HCT
February 2016Chow EJ, et al. Biol Blood Marrow Transplant – The Children’s Oncology Group (COG) has updated its Long-Term Follow-Up Guidelines for survivors of childhood, adolescent, and young adult cancer, including those undergoing autologous or allogeneic hematopoietic cell transplantation (HCT) and malignant and non-malignant diseases. -
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. -
Meeting the Needs of HCT Recipients Transitioning from Childhood to Adulthood
December 2015Cupit MC, et al. Bone Marrow Transplant – In this review, the authors use clinical vignettes to discuss the focused and specific survivorship needs of the growing pediatric and adolescent and young adult (AYA) populations after hematopoietic cell transplantation (HCT). -
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). -
Related, Unrelated HCT Outcomes Comparable in Pediatric ALL
March 2015Peters C, et al. J Clin Oncol — A large-scale, multi-center study of 411 transplants for children with high-risk acute lymphoblastic leukemia (ALL) has shown comparable transplant outcomes with sibling and unrelated donors. -
Autologous, Allogeneic HCT Both Effective in Pediatric High-Risk AML
February 2015Locatelli F, et al. Bone Marrow Transplant — A prospective multi-center study of 243 children with high-risk AML in CR1 undergoing allogeneic HCT (n=141) or autologous HCT (n=102), depending on availability of an HLA-compatible sibling donor. -
Review: How I Treat JMML
February 2015Locatelli F, et al. Blood — In this installment of the “How I treat ...” series in Blood, the authors outline the somatic or germline mutations that are the underlying cause of juvenile myelomonocytic leukemia (JMML) and describe their treatment algorithm for this aggressive hematopoietic disorder of infancy/early childhood. -
Similar Survival Rates in Children Transplanted with One vs. Two Umbilical Cord Blood Units
November 2014Wagner JE, et al. N Engl J Med – Children with high-risk blood cancers transplanted with one vs. two umbilical cord blood units had similar survival, according to a multi-center study of 224 cord blood transplants performed between 2006 and 2012 and reported to CIBMTR (Center for International Blood and Marrow Transplant Research). -
Long-Term Impact of TBI on Cognitive Functioning in Pediatric HCT
November 2014Willard VW, et al. J Clin Oncol — Using total-body irradiation (TBI) in pre-transplant conditioning regimens lowers cognitive ability, according to a study of 315 pediatric hematopoietic cell transplant (HCT) recipients. -
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. -
High Likelihood that Patients Needing Unrelated HCT Will Find Donor on Be The Match Registry
July 2014Gragert L, et al. N Engl J Med — A study of the U.S. marrow and cord blood registry using population-based genetic models for 21 race and ethnic groups has found that, depending on the group, between 66-97% of patients will have a human leukocyte antigen (HLA)-matched or minimally mismatched available adult donor on the Be The Match Registry®. -
Review: HCT and Novel Approaches in Pediatric ALL
July 2014Pulsipher MA, et al. Bone Marrow Transplant — In this review, the authors define the current best and promising future HCT practices for high-risk acute lymphoblastic leukemia (ALL) in children and adolescents and discuss the optimal timing for HCT in these patients. -
Review: How I Treat Relapsed AML in Children
June 2014Kaspers G. Brit J Haematol — This review discusses the practical clinical issues involved in the treatment of children with relapsed AML. -
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. -
Post-HCT Minimal Residual Disease Can Identify Impending Relapse in Pediatric ALL
March 2014Bader P, et al. BMT Tandem Meetings abstract oral presentation, February 2014 - Assessing post-transplant minimal residual disease can identify impending relapse in pediatric ALL patients who may therefore benefit from pre-emptive therapies, according to a prospective study of 115 patients. -
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. -
Childhood HCT Survivors Require Life-Long Monitoring for High-Risk Complications
December 2013Armenian S, et al. ASH abstract oral presentation, December 2013 – Pediatric hematopoietic cell transplant (HCT) recipients can experience high-risk health complications well into adulthood, and therefore require close monitoring, according to results of 317 pediatric HCT survivors participating in the Bone Marrow Transplant Survivor Study (BMTSS). -
Improved HCT Survival over Time for Adolescents and Young Adults
December 2013Wood WA, et al. ASH abstract oral presentation, December 2013 – Survival after HCT has improved significantly over time for adolescents and young adults (age 15-40 years), according to a large-scale, multi-center study of 1,244 myeloablative allogeneic transplants for ALL at U.S. centers over three time periods: 1990-1995, 1996-2001, and 2002-2007. -
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). -
Long-Term Follow-Up of HCT Recipients Made Easier
July 2013Majhail NS, et al. Bone Marrow Transplant - A summary of the updated screening and preventive practices guidelines for HCT recipients focusing on ways that health care providers can effectively integrate the guidelines into clinical practice. -
HCT Outcomes Comparable in High- and Standard-Risk Pediatric AML
April 2013Burke MJ, et al. Biol Blood Marrow Transplant - In this single-institution study of 50 consecutive pediatric patients (median age 14.9 years) transplanted for acute myeloid leukemia (AML) in first complete remission, high-risk patients and standard-risk patients had comparable survival.
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Myeloablative HCT Improves Survival in Younger Adults with ALL in First Remission
January 2013Gupta V, et al. Blood - A meta-analysis combining data from 13 studies including 2,962 adults has shown that allogeneic, but not autologous, hematopoietic cell transplantation (HCT) improves survival in younger adults with acute lymphoblastic leukemia (ALL) in first complete remission.