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Neurofibromatose

Neurofibromatose, also known as neurofibromatosis, designates a group of autosomal‑dominant genetic disorders characterized by the development of multiple benign nerve‑sheath tumors (neurofibromas) and a range of cutaneous, ocular, skeletal and neurologic manifestations. Three distinct clinical forms are recognized. Neurofibromatosis type 1 (NF1) results from mutations in the NF1 gene on chromosome 17 and is the most common, affecting roughly 1 in 3 000 individuals. Typical features include café‑au‑lait macules, axillary or inguinal freckling, Lisch iris hamartomas, and a predisposition to malignant peripheral nerve‑sheath tumors. Neurofibromatosis type 2 (NF2) is caused by mutations in the NF2 gene on chromosome 22 and is less frequent (about 1 in 25 000). Its hallmark is bilateral vestibular schwannomas, often leading to hearing loss, together with other cranial and spinal schwannomas. A third entity, schwannomatosis, involves multiple non‑vestibular schwannomas without the diagnostic criteria of NF1 or NF2 and is linked to mutations in the SMARCB1 or LZTR1 genes.

Diagnosis relies on clinical criteria supported by genetic testing. Imaging, particularly MRI, helps assess tumor burden

and
complications
such
as
spinal
cord
compression.
Management
is
multidisciplinary,
emphasizing
regular
surveillance,
surgical
excision
of
symptomatic
lesions,
and
targeted
therapies
(e.g.,
MEK
inhibitors
for
inoperable
plexiform
neurofibromas).
Pain
control,
orthopedic
intervention
and
visual
or
auditory
rehabilitation
are
often
required.
Life
expectancy
in
NF1
is
modestly
reduced
due
to
tumor‑related
complications;
NF2
and
schwannomatosis
carry
variable
prognoses
depending
on
tumor
location
and
growth.
Ongoing
research
focuses
on
molecular
pathways
to
develop
disease‑modifying
treatments.