December 2020. Volume 16. Number 4

Inoperable plexiform neurofibromas of neurofibromatosis may benefit from selumetinib

 
 
 
 
 
 
 
 
 
 
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AVC | Critically appraised articles

Gross AM, Wolters PL, Dombi E, Baldwin A, Whitcomb P, Fisher MJ, et al. Selumetinib in children with inoperable plexiform neurofibromas. N Engl J Med. 2020; 382:1430-42.

Reviewers: Aparicio Rodrigo M1, Aizpurua Galdeano P2.
1Pediatra. CS Entrevías. Profesor asociado. Facultad de Medicina. Universidad Complutense de Madrid. Madrid. España.
2Pediatra. CS Ondarreta. San Sebastián. Guipúzcoa. España.
Correspondence: María Aparicio Rodrigo. Email: maparicio@salud.madrid.org
Reception date: 13/09/2020
Acceptance date: 05/10/2020
Publication date: 21/10/2020

Abstract

Authors' conclusions: most children with neurofibromatosis type 1 and inoperable plexiform neurofibromas had durable tumor shrinkage and clinical benefit from selumetinib.

Reviewers ́ commentary: selumetinib could be effective in the treatment of plexiform neurofibromas of neurofibromatosis type 1 with complications, with a plateau in the evolution of the disease along with a decrease in the volume of neurofibromas of 20-30% in half of those treated and a mild-moderate clinical improvement of other symptoms such as pain or functional impotence. This is a medication to be considered in the future in the treatment of neurofibromatosis type 1 (NF1) since it is a disease with no other possible treatment and its symptoms have a significant impact on the quality of life of the patient.

How to cite this article

Aparicio Rodrigo M, Aizpurua Galdeano P. Los neurofibromas plexiformes inoperables de la neurofibromatosis podrían mejorar con selumetinib. Evid Pediatr. 2020;16:45.

AVC | Critically appraised articles

Gross AM, Wolters PL, Dombi E, Baldwin A, Whitcomb P, Fisher MJ, et al. Selumetinib in children with inoperable plexiform neurofibromas. N Engl J Med. 2020; 382:1430-42.

Reviewers: Aparicio Rodrigo M1, Aizpurua Galdeano P2.
1Pediatra. CS Entrevías. Profesor asociado. Facultad de Medicina. Universidad Complutense de Madrid. Madrid. España.
2Pediatra. CS Ondarreta. San Sebastián. Guipúzcoa. España.
Correspondence: María Aparicio Rodrigo. Email: maparicio@salud.madrid.org
Reception date: 13/09/2020
Acceptance date: 05/10/2020
Publication date: 21/10/2020

How to cite this article

Aparicio Rodrigo M, Aizpurua Galdeano P. Los neurofibromas plexiformes inoperables de la neurofibromatosis podrían mejorar con selumetinib. Evid Pediatr. 2020;16:45.

21/10/2020

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