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Published on: May 27, 2011
Survival Trends in Infants Undergoing Allogeneic Hematopoietic Cell Transplant
Suhag H Parikh1, Prakash Satwani2, Kwang Woo Ahn3
1Department of Pediatric Blood and Marrow Transplant, Duke University Medical Center, Durham, North Carolina.
Insights
Survival rates for infants undergoing allogeneic hematopoietic cell transplant have not improved for those with malignant conditions. While survival improved for nonmalignant conditions initially, significant challenges with relapse and toxicity persist.
Area of Science:
- Hematology
- Pediatric Oncology
- Transplant Immunology
Background:
- Allogeneic hematopoietic cell transplant (HCT) offers survival benefits but data for infants are limited.
- Infants, particularly those with malignant conditions, are often excluded from HCT studies.
- Understanding survival trends in infants undergoing HCT is crucial for improving outcomes.
Purpose of the Study:
- To analyze overall survival (OS) trends in infants receiving allogeneic HCT.
- To evaluate disease relapse and toxicity rates in infant HCT recipients.
- To identify factors influencing outcomes in pediatric HCT.
Main Methods:
- A multicenter cohort study using data from the Center for International Blood and Marrow Transplant Research (CIBMTR).
- Time-trend analysis of three periods: 2000-2004, 2005-2009, and 2010-2014.
- Inclusion of infants (≤1 year) with malignant and nonmalignant conditions.
Main Results:
- Overall survival (OS) did not improve for infants with malignant conditions over the 15-year period.
- Survival rates improved for infants with nonmalignant diseases from 2000-2004 but plateaued thereafter.
- Relapse rates increased in the malignant cohort, and sinusoidal obstruction syndrome was a significant toxicity for all infants.
Conclusions:
- Survival outcomes for infants with malignant conditions post-allogeneic HCT remain suboptimal.
- Improved donor and graft selection, alongside strategies to mitigate relapse and toxicity, are needed.
- Further research focusing on infant-specific HCT protocols is warranted.
Importance:
Studies demonstrating improved survival after allogeneic hematopoietic cell transplant generally exclude infants.
Objective:
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.
Exposures:
Allogeneic hematopoietic cell transplant.
Main Outcomes And Measures:
Survival trends, disease relapse, and toxicity.
Results:
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.
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