PMID- 38443692
OWN - NLM
STAT- Publisher
LR  - 20240305
IS  - 1573-7373 (Electronic)
IS  - 0167-594X (Linking)
DP  - 2024 Mar 5
TI  - Cutaneous toxicities of mitogen-activated protein kinase inhibitors in children 
      and young adults with neurofibromatosis-1.
LID - 10.1007/s11060-024-04617-2 [doi]
AB  - PURPOSE: Neurofibromatosis type 1 (NF1) is an autosomal dominant disorder which 
      commonly causes neoplasms leading to disfigurement or dysfunction. 
      Mitogen-activated protein kinase inhibitors (MEKi) are generally well-tolerated 
      treatments which target neural tumor progression in patients with NF1. However, 
      cutaneous adverse events (CAEs) are common and may hinder patients' abilities to 
      remain on treatment, particularly in children. We aim to characterize CAEs 
      secondary to MEKi treatment in pediatric and young adult patients with NF1. 
      METHODS: We reviewed institutional medical records of patients under 30 years 
      with a diagnosis of "NF1," "NF2," or "other neurofibromatoses" on MEKi therapy 
      between January 1, 2019 and June 1, 2022. We recorded the time-to-onset, type, 
      and distribution of CAEs, non-cutaneous adverse events (AEs), AE management, and 
      tumor response. RESULTS: Our cohort consisted of 40 patients with NF1 (median 
      age, 14 years). Tumor types included low-grade gliomas (51%) and plexiform 
      neurofibromas (38%). MEKi used included selumetinib (69%), trametinib (25%), and 
      mirdametinib (6%). A total of 74 CAEs occurred, with 28 cases of acneiform rash 
      (38%). Other common CAEs were paronychia, seborrheic dermatitis, eczema, xerosis, 
      and oral mucositis. The most common treatments included oral antibiotics and 
      topical corticosteroids. Most patients had clinical (stable or improved) tumor 
      response (71%) while 29% had tumor progression while on a MEKi. There was no 
      significant association between CAE presence and tumor response (p = 0.39). 
      CONCLUSIONS: Improvement in characterization of MEKi toxicities and their 
      management is important to develop treatment guidelines for pediatric and young 
      adult patients with NF1 on MEKi therapy.
CI  - (c) 2024. The Author(s), under exclusive licence to Springer Science+Business 
      Media, LLC, part of Springer Nature.
FAU - Peacock, Brianna C
AU  - Peacock BC
AUID- ORCID: 0000-0003-1865-9105
AD  - Texas A&M University School of Engineering Medicine, Houston, TX, USA.
FAU - Tripathy, Sanjna
AU  - Tripathy S
AUID- ORCID: 0000-0002-9728-1042
AD  - McGovern Medical School, The University of Texas Health Sciences Center, Houston, 
      TX, USA.
FAU - Hanania, Hannah L
AU  - Hanania HL
AUID- ORCID: 0000-0002-6155-2226
AD  - Baylor College of Medicine, Houston, TX, USA.
FAU - Wang, Hannah Y
AU  - Wang HY
AD  - Baylor College of Medicine, Houston, TX, USA.
FAU - Sadighi, Zsila
AU  - Sadighi Z
AUID- ORCID: 0000-0003-1686-7760
AD  - Department of Pediatrics, The University of Texas MD Anderson Cancer Center, 
      Houston, TX, USA.
FAU - Patel, Anisha B
AU  - Patel AB
AUID- ORCID: 0000-0002-5372-784X
AD  - Department of Dermatology, Internal Medicine, The University of Texas MD Anderson 
      Cancer Center, 1515 Holcombe Blvd, Box 1452, Houston, TX, 77030, USA. 
      apatel11@mdanderson.org.
LA  - eng
PT  - Journal Article
DEP - 20240305
PL  - United States
TA  - J Neurooncol
JT  - Journal of neuro-oncology
JID - 8309335
SB  - IM
OTO - NOTNLM
OT  - Child
OT  - Mitogen-activated protein kinases
OT  - Neurofibromatoses
OT  - Retrospective studies
OT  - Skin diseases
EDAT- 2024/03/06 00:42
MHDA- 2024/03/06 00:42
CRDT- 2024/03/05 23:32
PHST- 2024/01/24 00:00 [received]
PHST- 2024/02/21 00:00 [accepted]
PHST- 2024/03/06 00:42 [medline]
PHST- 2024/03/06 00:42 [pubmed]
PHST- 2024/03/05 23:32 [entrez]
AID - 10.1007/s11060-024-04617-2 [pii]
AID - 10.1007/s11060-024-04617-2 [doi]
PST - aheadofprint
SO  - J Neurooncol. 2024 Mar 5. doi: 10.1007/s11060-024-04617-2.