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Cumulative Hematologic Morbidity and Outcomes in Shwachman–Diamond Syndrome

Kasiani C. Myers, Jean Donadieu, Benjamin Goldberg, Manon Delafoy, Cornelia Zeidler, Sabine Mellor-Heineke, Yang Wan, Xiaofan Zhu, Simone Cesaro, Marco Cipolli, 渡邊 健一郎, Lucy C. Fox, Caroline Rutten, Taco W. Kuijpers, Antonis Kattamis, Polyxeni Delaporta, Ulla Wartiovaara‐Kautto, Outi Kilpivaara, Alison A. Bertuch, Candelaria O’Farrell, Melanie M. Cotter, Eoghan Dunlea, Christopher R. Reilly, Alan J. Warren, Amy Geddis, Edie Weller, Akiko Shimamura

Peer-reviewed journalReal-world use

In the authors' words

This journal does not let us republish the full abstract. Here are the two sentences Pipette selected, quoted from it. Read the rest at the publisher.

Main result
In an international cohort of 865 patients, Shwachman–Diamond syndrome was associated with a high risk of myeloid cancer and poor survival.
Limitation the authors admit
Early hematopoietic stem-cell transplantation for high-risk features had a potential benefit.
Read the paper

Appeared: Friday, September 25. New England Journal of Medicine. Peer-reviewed journal.

DOI: 10.1056/nejmc2605001

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