Have survival rates improved for infants 1 year or younger who received allogeneic hematopoietic cell transplantation (HCT)?
This cohort study evaluated 2498 infants who received HCT. For infants with nonmalignant diseases, survival rates improved from 2000 through 2004 to 2005 through 2009 and stabilized after this time; for infants with malignant conditions, survival did not improve from 2000 through 2014.
Although children and adults who receive HCT are surviving longer than before, infants who need HCT face unique challenges; research to enhance survival should focus on optimizing donor selection, refining conditioning regimens, reducing toxicity, and improving supportive care.
Studies demonstrating improved survival after allogeneic hematopoietic cell transplant generally exclude infants.
To analyze overall survival trends and other outcomes among infants who undergo allogeneic hematopoietic cell transplant.
Design, Setting, and Participants
In this cohort study, we used time-trend analysis to evaluate 3 periods: 2000 through 2004, 2005 through 2009, and 2010 through 2014. The study was conducted in a multicenter setting through the Center for International Blood and Marrow Transplant Research, which is made up of a voluntary working group of more than 450 transplant centers worldwide. Two groups of infants aged 1 year or younger in 2 cohorts were included: those with malignant conditions, such as leukemia, and those with nonmalignant disorders, including immunodeficiencies. Data analysis was conducted from July 2017 to December 2018.
Allogeneic hematopoietic cell transplant.
Main Outcomes and Measures
Survival trends, disease relapse, and toxicity.
A total of 2498 infants with a median age of 7 months (range, <1-12 months) were included. In the nonmalignant cohort (n = 472), survival rates improved from the first to the second period (hazard ratio, 0.77 [95% CI, 0.63-0.93]; P = .007) but did not change after 2004. Compared with infants with nonmalignant diseases (n = 2026; 3-year overall survival: 2000-2004, 375/577 [65.0%]; 2005-2009, 503/699 [72.0%]; and 2010-2014, 555/750 [74.0%]), those with malignant conditions had poorer survival rates, without improvement over time (3-year overall survival: 2000-2004, 109/199 [54.8%]; 2005-2009, 104/161 [64.6%]; and 2010-2014, 66/112 [58.9%]). From 2000 through 2014, relapse rates increased in infants with malignant conditions (3-year relapse rate: 2000-2004, 19% [95% CI, 14%-25%]; 2005-2009, 23% [95% CI, 17%-30%]; 2010-2014, 36% [95% CI, 27%-46%]; P = .01). Sinusoidal obstruction syndrome was frequent, occurring with a cumulative incidence of 13% (95% CI, 11%-16%) of infants with nonmalignant diseases and 32% (95% CI, 22%-42%) of those with malignant diseases. Generally, recipients of human leukocyte antigen–identical sibling bone marrow grafts had the best outcomes.
Conclusions and Relevance
Survival rates have not improved for infants with malignant diseases over the 15-year study period. Infants with nonmalignant diseases had improved survival rates in the earlier but not the later study period. Higher relapses for the malignant cohort and toxicities for all infants remain significant challenges. Strategies to reduce relapse and toxicity and optimize donor and graft selection may improve outcomes in the future.
Parikh SH, Satwani P, Ahn KW, et al. Survival Trends in Infants Undergoing Allogeneic Hematopoietic Cell Transplant. JAMA Pediatr. Published online May 01, 2019173(5):e190081. doi:10.1001/jamapediatrics.2019.0081
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