Segmental schwannomatosis: characteristics in 12 patients.


Journal

Orphanet journal of rare diseases
ISSN: 1750-1172
Titre abrégé: Orphanet J Rare Dis
Pays: England
ID NLM: 101266602

Informations de publication

Date de publication:
22 08 2019
Historique:
received: 21 03 2019
accepted: 13 08 2019
entrez: 24 8 2019
pubmed: 24 8 2019
medline: 1 7 2020
Statut: epublish

Résumé

Segmental schwannomatosis is characterized by multiple schwannomas affecting one-limb or less than 5 contiguous segments of spine. Its characteristics are not well described in the literature. Our objective was to better describe the demographic and clinical characteristics of this condition. This was a retrospective, bi-center study conducted in two French expert centers for neurofibromatosis and schwannomatosis. The clinical, radiographic, pathological and molecular aspects were extracted from patients' clinical records. Twelve patients with segmental schwannomatosis were identified. Eight were female and 4 were male. The median age at initial symptom was 29 years (range: 6-60 years) and the median age at diagnosis was 34.5 years (range: 13-65 years). Pain was the initial symptom for the majority of patients (7 of 12). The number of tumors was variable with six patients having more than 10 tumors. Peripheral distribution was seen in all patients. Quality of life could be impaired (median Dermatology Life Quality Index score was 4.5 (range: 2-13). The median duration of follow up was 3 years (range: 1-26). Chronic pain was the main complication (9 of 12 patients). Surgical intervention to control chronic pain was performed for 9 patients of whom 5 experienced recurrence of tumors. Molecular investigations revealed heterozygous LZTR1 variants in 3 of 9 patients. Segmental schwannomatosis is a rare condition that may start early in life and often remains undiagnosed for many years. Pain is the main symptom and consequently could impair the quality of life. Surgery seems to be effective, but recurrences are frequent. Some patients carried heterozygous LZTR1 variants. Further studies are needed to better understand this rare condition.

Sections du résumé

BACKGROUND
Segmental schwannomatosis is characterized by multiple schwannomas affecting one-limb or less than 5 contiguous segments of spine. Its characteristics are not well described in the literature. Our objective was to better describe the demographic and clinical characteristics of this condition.
METHODS
This was a retrospective, bi-center study conducted in two French expert centers for neurofibromatosis and schwannomatosis. The clinical, radiographic, pathological and molecular aspects were extracted from patients' clinical records.
RESULTS
Twelve patients with segmental schwannomatosis were identified. Eight were female and 4 were male. The median age at initial symptom was 29 years (range: 6-60 years) and the median age at diagnosis was 34.5 years (range: 13-65 years). Pain was the initial symptom for the majority of patients (7 of 12). The number of tumors was variable with six patients having more than 10 tumors. Peripheral distribution was seen in all patients. Quality of life could be impaired (median Dermatology Life Quality Index score was 4.5 (range: 2-13). The median duration of follow up was 3 years (range: 1-26). Chronic pain was the main complication (9 of 12 patients). Surgical intervention to control chronic pain was performed for 9 patients of whom 5 experienced recurrence of tumors. Molecular investigations revealed heterozygous LZTR1 variants in 3 of 9 patients.
CONCLUSION
Segmental schwannomatosis is a rare condition that may start early in life and often remains undiagnosed for many years. Pain is the main symptom and consequently could impair the quality of life. Surgery seems to be effective, but recurrences are frequent. Some patients carried heterozygous LZTR1 variants. Further studies are needed to better understand this rare condition.

Identifiants

pubmed: 31438995
doi: 10.1186/s13023-019-1176-4
pii: 10.1186/s13023-019-1176-4
pmc: PMC6704629
doi:

Substances chimiques

LZTR1 protein, human 0
Transcription Factors 0

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

207

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Auteurs

Abdulqader Alaidarous (A)

Service de Dermatologie, Hôpital Larrey, CHU de Toulouse, Toulouse, France. abdulqader.alaidarous@gmail.com.

Beatrice Parfait (B)

Service de Génétique et Biologie Moléculaires, Hôpital Cochin, Assistance Publique-Hôpitaux de Paris, Paris, France.
UMR INSERM 1016 Institut Cochin & Université Paris Descartes, Sorbonne Paris Cité, Paris, France.

Salah Ferkal (S)

Service de Dermatologie, Hôpital Henri Mondor, Assistance Publique-Hôpitaux de Paris, Creteil, France.

Joëlle Cohen (J)

Service de Génétique et Biologie Moléculaires, Hôpital Cochin, Assistance Publique-Hôpitaux de Paris, Paris, France.

Pierre Wolkenstein (P)

Service de Dermatologie, Hôpital Henri Mondor, Assistance Publique-Hôpitaux de Paris, Creteil, France.

Juliette Mazereeuw-Hautier (J)

Service de Dermatologie, Hôpital Larrey, CHU de Toulouse, Toulouse, France.

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Classifications MeSH