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Survival rates after HSCT have not improved among infants with malignant diseases

Friday, March 22, 2019
Suhag Parikh

Infants with malignant diseases who underwent allogeneic hematopoietic stem cell transplantation did not demonstrate improved survival rates over a 15-year period, according to results of a study in JAMA Pediatrics.

Survival rates among infants with nonmalignant diseases who received allogeneic HSCT improved only during the earlier part of the study period, results showed.

Past studies of survival outcomes following HSCT have not analyzed changes over time among infants, who may be at greater risk for toxicities and transplant-related complications, according to researchers.

“Overall survival outcomes after allogeneic [HSCT] have improved over time: patients who received transplants in 2003 through 2007 experienced a 41% reduction in the hazard of death from any cause and a 52% reduction in transplant-related mortality compared with those diagnosed in 1993 through 1997,” Suhag H. Parikh, MD, pediatric bone marrow transplant specialist at Duke University Hospital, and colleagues wrote. “A similar decrease in transplant-related mortality was noted in patients with acute myeloid leukemia who were younger than 50 years. However, these studies did not focus on infants, who receive a small portion of overall transplants performed annually.”

In the multicenter cohort study, Parikh and colleagues evaluated 2,498 infants aged 1 year and younger (median age, 7 months; 65.3% white) who received their first allogeneic HSCT at transplant centers involved with the Center for International Blood and Marrow Transplant Research, a working group of more than 450 transplant centers around the world. Researchers grouped infants according to whether they had a malignant diagnosis (n = 472) — such as acute lymphoblastic leukemia (n = 182) — or a nonmalignant condition (n = 2,026), such as severe combined immunodeficiency (n = 834). Infants with malignant diagnoses tended to be older (87% aged 6 to 12 months).

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