{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,14]],"date-time":"2026-09-14T21:10:52Z","timestamp":1789420252979,"version":"build-2803163510"},"reference-count":25,"publisher":"American Academy of Pediatrics (AAP)","issue":"6","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2014,6,1]]},"abstract":"<jats:p>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.<\/jats:p>","DOI":"10.1542\/peds.2013-1224","type":"journal-article","created":{"date-parts":[[2014,5,27]],"date-time":"2014-05-27T01:34:37Z","timestamp":1401154477000},"page":"e1792-e1797","source":"Crossref","is-referenced-by-count":28,"title":["Sirolimus Improves Pain in NF1 Patients With Severe Plexiform Neurofibromas"],"prefix":"10.1542","volume":"133","author":[{"given":"Camille","family":"Hua","sequence":"first","affiliation":[{"name":"AP-HP, H\u00f4pital Henri-Mondor, Service de Dermatologie, Centre de r\u00e9f\u00e9rence des Neurofibromatoses, Cr\u00e9teil, France;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ouidad","family":"Zehou","sequence":"additional","affiliation":[{"name":"AP-HP, H\u00f4pital Henri-Mondor, Service de Dermatologie, Centre de r\u00e9f\u00e9rence des Neurofibromatoses, Cr\u00e9teil, France;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"St\u00e9phane","family":"Ducassou","sequence":"additional","affiliation":[{"name":"CHU Bordeaux, GH Pellegrin, Service d'Onco-H\u00e9matologie P\u00e9diatrique, Bordeaux, France;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"V\u00e9ronique","family":"Minard-Colin","sequence":"additional","affiliation":[{"name":"Institut Gustave Roussy, D\u00e9partement de Canc\u00e9rologie de l'Enfant et de l'Adolescent, Villejuif, France;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dominique","family":"Hamel-Teillac","sequence":"additional","affiliation":[{"name":"H\u00f4pital Priv\u00e9 d\u2019Antony, Service de Dermatologie P\u00e9diatrique, Antony, France; and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pierre","family":"Wolkenstein","sequence":"additional","affiliation":[{"name":"AP-HP, H\u00f4pital Henri-Mondor, Service de Dermatologie, Centre de r\u00e9f\u00e9rence des Neurofibromatoses, Cr\u00e9teil, France;"},{"name":"LIC EA 4393, H\u00f4pital Henri Mondor, Assistance Publique H\u00f4pitaux de Paris, Universit\u00e9 Paris-Est Cr\u00e9teil, Cr\u00e9teil, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Laurence","family":"Valeyrie-Allanore","sequence":"additional","affiliation":[{"name":"AP-HP, H\u00f4pital Henri-Mondor, Service de Dermatologie, Centre de r\u00e9f\u00e9rence des Neurofibromatoses, Cr\u00e9teil, France;"},{"name":"LIC EA 4393, H\u00f4pital Henri Mondor, Assistance Publique H\u00f4pitaux de Paris, Universit\u00e9 Paris-Est Cr\u00e9teil, Cr\u00e9teil, France"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"417","published-online":{"date-parts":[[2014,6,1]]},"reference":[{"issue":"11","key":"2021121015590124600_B1","doi-asserted-by":"crossref","first-page":"704","DOI":"10.1136\/jmg.26.11.704","article-title":"A genetic study of von Recklinghausen neurofibromatosis in south east Wales. 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