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American Academy of Pediatrics
Case Report

Sirolimus Improves Pain in NF1 Patients With Severe Plexiform Neurofibromas

Camille Hua, Ouidad Zehou, Stéphane Ducassou, Véronique Minard-Colin, Dominique Hamel-Teillac, Pierre Wolkenstein and Laurence Valeyrie-Allanore
Pediatrics June 2014, 133 (6) e1792-e1797; DOI: https://doi.org/10.1542/peds.2013-1224
Camille Hua
aAP-HP, Hôpital Henri-Mondor, Service de Dermatologie, Centre de référence des Neurofibromatoses, Créteil, France;
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Ouidad Zehou
aAP-HP, Hôpital Henri-Mondor, Service de Dermatologie, Centre de référence des Neurofibromatoses, Créteil, France;
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Stéphane Ducassou
bCHU Bordeaux, GH Pellegrin, Service d'Onco-Hématologie Pédiatrique, Bordeaux, France;
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Véronique Minard-Colin
cInstitut Gustave Roussy, Département de Cancérologie de l'Enfant et de l'Adolescent, Villejuif, France;
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Dominique Hamel-Teillac
dHôpital Privé d’Antony, Service de Dermatologie Pédiatrique, Antony, France; and
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Pierre Wolkenstein
aAP-HP, Hôpital Henri-Mondor, Service de Dermatologie, Centre de référence des Neurofibromatoses, Créteil, France;
eLIC EA 4393, Hôpital Henri Mondor, Assistance Publique Hôpitaux de Paris, Université Paris-Est Créteil, Créteil, France
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Laurence Valeyrie-Allanore
aAP-HP, Hôpital Henri-Mondor, Service de Dermatologie, Centre de référence des Neurofibromatoses, Créteil, France;
eLIC EA 4393, Hôpital Henri Mondor, Assistance Publique Hôpitaux de Paris, Université Paris-Est Créteil, Créteil, France
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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.

  • neurofibromatosis type 1
  • plexiform neurofibromas
  • therapeutics
  • pain management
  • sirolimus
  • Accepted January 29, 2014.
  • Copyright © 2014 by the American Academy of Pediatrics

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Pediatrics
Vol. 133, Issue 6
1 Jun 2014
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Sirolimus Improves Pain in NF1 Patients With Severe Plexiform Neurofibromas
Camille Hua, Ouidad Zehou, Stéphane Ducassou, Véronique Minard-Colin, Dominique Hamel-Teillac, Pierre Wolkenstein, Laurence Valeyrie-Allanore
Pediatrics Jun 2014, 133 (6) e1792-e1797; DOI: 10.1542/peds.2013-1224

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Sirolimus Improves Pain in NF1 Patients With Severe Plexiform Neurofibromas
Camille Hua, Ouidad Zehou, Stéphane Ducassou, Véronique Minard-Colin, Dominique Hamel-Teillac, Pierre Wolkenstein, Laurence Valeyrie-Allanore
Pediatrics Jun 2014, 133 (6) e1792-e1797; DOI: 10.1542/peds.2013-1224
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