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Sirolimus improves pain in NF1 patients with severe plexiform neurofibromas
- Source :
- Pediatrics. 133(6)
- Publication Year :
- 2014
-
Abstract
- Plexiform neurofibromas (PNs) are common and potentially debilitating complications of neurofibromatosis 1 (NF1). These benign nerve-sheath tumors are associated with significant pain and morbidity because they compress vital structures. The mammalian target of rapamycin (mTOR) pathway is a major mediator involved in tumor growth in NF1. We present 3 cases of patients with NF1, aged 8, 16, and 17 years, followed for inoperable and symptomatic PNs; patients received sirolimus for life-threatening and painful neurofibromas after multidisciplinary consultation. Epidemiologic, clinical, and radiologic data were retrospectively collected. The volume of PNs did not differ between baseline and 12-month follow-up and pain was alleviated, with withdrawal of analgesics in 2 cases at 6 months, and significantly decreased for the third case. Sirolimus for inoperable symptomatic PNs in patients with NF1 permitted stabilization of mass and produced unpredictable and important alleviation of pain in all cases with good tolerance. This treatment was proposed in extreme cases, in absence of therapeutic alternatives, after multidisciplinary consensus. The mTOR pathway may be both a major mediator of NF1 tumor growth and regulator of nociceptor sensitivity. mTOR inhibitors clinically used as anticancer and immunosuppressant drugs could be a potential treatment of chronic pain.
- Subjects :
- Oncology
Drug
Male
medicine.medical_specialty
Neurofibromatosis 1
Adolescent
media_common.quotation_subject
Drug Administration Schedule
Plexiform neurofibroma
Internal medicine
medicine
Neurofibroma
Humans
Neurofibromatosis
Cooperative Behavior
Child
PI3K/AKT/mTOR pathway
media_common
Pain Measurement
Neurofibroma, Plexiform
Sirolimus
Dose-Response Relationship, Drug
business.industry
Chronic pain
medicine.disease
Surgery
Abdominal Neoplasms
Pediatrics, Perinatology and Child Health
Nociceptor
Female
Interdisciplinary Communication
Chronic Pain
business
medicine.drug
Follow-Up Studies
Subjects
Details
- ISSN :
- 10984275
- Volume :
- 133
- Issue :
- 6
- Database :
- OpenAIRE
- Journal :
- Pediatrics
- Accession number :
- edsair.doi.dedup.....605cc257a8864f92a2c615a4e9617455