Hematopoietic cell transplantation using treosulfan-based conditioning for IL-10 receptor deficiency

Madhavi Lakkaraja, Erica G. Schmitt, Amika K. Sood, Karin Chen, Arunkumar J. Modi, James W. Verbsky, Troy Torgerson, Ghassan Wahbeh, Aleksandra Petrovic, K. Scott Baker, Megan A. Cooper, Lauri M. Burroughs · 2027

Read Hematopoietic cell transplantation using treosulfan-based conditioning for IL-10 receptor deficiency by Madhavi Lakkaraja, Erica G. Schmitt, Amika K. Sood, Karin Chen, Arunkumar J. Modi, James W. Verbsky, Troy Torgerson, Ghassan Wahbeh, Aleksandra Petrovic, K. Scott Baker, Megan A. Cooper, Lauri M. Burroughs (2027) in the Tower of Babel, a free online library of public-domain works preserved in clean, searchable text. No sign-up. The full text is served as a plain-text file at a predictable URL, where text is present: https://antarctic-labs.com/tower-of-babel/pmc-13671123.txt.

Description

Lakkaraja and colleagues report in 2027 two patients transplanted for IL-10 receptor deficiency, an inherited immune defect that causes severe bowel disease in infancy. Standard immune drugs control it poorly, and anakinra can calm it but not cure it. Both patients received blood stem cell transplants prepared with treosulfan-based conditioning. The approach gave full donor engraftment and excellent survival.

The two cases span nearly a decade, showing the method's durability. The report also confirms new disease-causing gene variants. The authors highlight anakinra as an effective bridge that holds the disease until transplant can proceed.

Catalog record

CREATORMadhavi Lakkaraja, Erica G. Schmitt, Amika K. Sood, Karin Chen, Arunkumar J. Modi, James W. Verbsky, Troy Torgerson, Ghassan Wahbeh, Aleksandra Petrovic, K. Scott Baker, Megan A. Cooper, Lauri M. Burroughs
YEAR2027
COLLECTIONPAPERS
FORMATTXT
FILE SIZE12 KB
VERSION1.0
LICENSECC BY 4.0
RIGHTSCC BY
SOURCEEurope PMC

Source: https://europepmc.org/articles/PMC13671123

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