Selumetinib in the treatment of plexiform neurofibromas in neurofibromatosis type 1: a 6-year prospective case series with review of the literature
Francesco Baldo, Luca Messana, Alberto Andresani, Andrea Magnolato, Flora Maria Murru, Anita Spezzacatene, Elisabetta Cattaruzzi, Alessandra Maestro, Gilda Paternuosto, Davide Zanon, Laura Diplotti, Paola Michieletto, Egidio Barbi, Irene Bruno · 2026
Read Selumetinib in the treatment of plexiform neurofibromas in neurofibromatosis type 1: a 6-year prospective case series with review of the literature by Francesco Baldo, Luca Messana, Alberto Andresani, Andrea Magnolato, Flora Maria Murru, Anita Spezzacatene, Elisabetta Cattaruzzi, Alessandra Maestro, Gilda Paternuosto, Davide Zanon, Laura Diplotti, Paola Michieletto, Egidio Barbi, Irene Bruno (2026) 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-13474747.txt.
Description
Baldo and co-authors followed 23 patients with neurofibromatosis type 1 treated with selumetinib. It is the first drug shown to shrink plexiform neurofibromas, the congenital nerve tumors that disfigure roughly half of people with the condition. Their 2026 prospective case series from a Trieste children's institute covers treatment from January 2017 to June 2023, comparing tumor volumes on MRI before and during therapy.
Over a mean follow-up of 1,085 days, thirteen patients, 57% of the cohort, shrank their tumors by more than 20% in volume, seven held stable, and three grew despite treatment. The median volumetric reduction across patients was 23% against baseline scans. The authors call the MEK inhibitor useful and safe in real-world use, while flagging the open question of what to offer the minority who never respond or stop responding.
Catalog record
| CREATOR | Francesco Baldo, Luca Messana, Alberto Andresani, Andrea Magnolato, Flora Maria Murru, Anita Spezzacatene, Elisabetta Cattaruzzi, Alessandra Maestro, Gilda Paternuosto, Davide Zanon, Laura Diplotti, Paola Michieletto, Egidio Barbi, Irene Bruno |
|---|---|
| YEAR | 2026 |
| COLLECTION | PAPERS |
| FORMAT | TXT |
| FILE SIZE | 29 KB |
| VERSION | 1.0 |
| LICENSE | CC BY 4.0 |
| RIGHTS | CC BY |
| SOURCE | Europe PMC |