Early HCT Boosts Survival in Youth With SDS
A study published in The New England Journal of Medicine suggests that early hematopoietic cell transplants can improve long-term survival for children with Shwachman-Diamond Syndrome. Researchers recommend intervening before the onset of life-threatening malignancies like leukemia.
Why it matters
This shift in clinical protocol could significantly increase the life expectancy of patients with this rare genetic condition by moving from reactive to proactive treatment.
More than 90% of children born with the rare bone marrow condition called Shwachman-Diamond Syndrome (SDS) survive beyond age 20. But fewer than 30% live beyond age 50.
However, longer-term survival could be improved by intervening upon earlier signs of high risk, according to a study published Sept. 23, 2026 , in The New England Journal of Medicine.
The study was co-led by Kasiani Myers, MD , Division of Bone Marrow Transplantation and Immune Deficiency at Cincinnati Children's. Co-authors included 26 fellow researchers involved in the International SDS Research Consortium.
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