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. -
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. -
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.