Neurofibromatosis type 1 medical therapy: Difference between revisions
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__NOTOC__ | __NOTOC__ | ||
{{Neurofibromatosis type 1}} | {{Neurofibromatosis type 1}} | ||
{{CMG}}; {{AE}} | {{CMG}}; {{AE}}[[User:MoisesRomo|Moises Romo M.D.]] | ||
==Overview== | ==Overview== | ||
There is no treatment for [ | There is no treatment for [[neurofibromatosis type 1]]; the mainstay of therapy is supportive care. [[Therapy]] for a patient with [[neurofibromatosis type 1]] is aimed at decreasing [[symptoms]] and improving [[quality of life]]. At every [[age]], different problems may develop, so a specific approach is necessary for the age and presentation of the individual. Supportive therapy for [[neurofibromatosis type 1]] includes pain relief, [[psychotherapy]], and [[antidepressants]]. | ||
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==Medical Therapy== | ==Medical Therapy== | ||
* | *At every age, different problems may appear, so a specific approach is necessary for the age and presentation of the individual.<ref name=":0">{{cite journal|doi=10.1016/B978-0-444-62702-5.00004-4.}}</ref> | ||
*Supportive therapy for [[neurofibromatosis type 1]] includes [[pain]] relief, [[psychotherapy]], and [[antidepressants]].<ref name="ListernickCharrow1989">{{cite journal|last1=Listernick|first1=Robert|last2=Charrow|first2=Joel|last3=Greenwald|first3=Mark J.|last4=Esterly|first4=Nancy B.|title=Optic gliomas in children with neurofibromatosis type 1|journal=The Journal of Pediatrics|volume=114|issue=5|year=1989|pages=788–792|issn=00223476|doi=10.1016/S0022-3476(89)80137-4}}</ref> | |||
*[[Stimulant]] [[medications]] should be considered for patients with [[Attention deficit hyperactivity disorder|attention deficitis]] (ADHD).<ref name="MautnerKluwe2010">{{cite journal|last1=Mautner|first1=V.-F.|last2=Kluwe|first2=L.|last3=Friedrich|first3=R. E.|last4=Roehl|first4=A. C.|last5=Bammert|first5=S.|last6=Hogel|first6=J.|last7=Spori|first7=H.|last8=Cooper|first8=D. N.|last9=Kehrer-Sawatzki|first9=H.|title=Clinical characterisation of 29 neurofibromatosis type-1 patients with molecularly ascertained 1.4 Mb type-1 NF1 deletions|journal=Journal of Medical Genetics|volume=47|issue=9|year=2010|pages=623–630|issn=0022-2593|doi=10.1136/jmg.2009.075937}}</ref> | |||
*[[Ketotifen]] ([[mast cell]] blocker that produces its [[depigmentation]]) has been proposed to treat some of the [[symptoms]] of [[pruritus]], [[pain]], and [[muscle weakness]] associated with [[neurofibromatosis type 1]]. The dose of [[ketotifen]] is 2 to 4 milligrams daily.<ref name="pmid8481017">{{cite journal |vauthors=Riccardi VM |title=A controlled multiphase trial of ketotifen to minimize neurofibroma-associated pain and itching |journal=Arch Dermatol |volume=129 |issue=5 |pages=577–81 |date=May 1993 |pmid=8481017 |doi= |url=}}</ref><ref name="urlscielo.isciii.es">{{cite web |url=http://scielo.isciii.es/pdf/odonto/v21n5/original1.pdf |title=scielo.isciii.es |format= |work= |accessdate=}}</ref> | |||
*[[Chemotherapy]] may be necessary for certain [[tumors]].<ref name=":0" /> Carboplatin-based [[chemotherapy]] combined with [[vincristine]], is the preferred therapy for [[neurofibromatosis type 1]] associated low-grade [[gliomas]].<ref name="pmid26235348">{{cite journal |vauthors=Dodgshun AJ, Maixner WJ, Heath JA, Sullivan MJ, Hansford JR |title=Single agent carboplatin for pediatric low-grade glioma: A retrospective analysis shows equivalent efficacy to multiagent chemotherapy |journal=Int. J. Cancer |volume=138 |issue=2 |pages=481–8 |date=January 2016 |pmid=26235348 |doi=10.1002/ijc.29711 |url=}}</ref> | |||
*[[Bevacizumab]] has demonstrated good response on vision testing in cases of refractory [[optic pathway glioma]].<ref name="pmid19165892">{{cite journal |vauthors=Packer RJ, Jakacki R, Horn M, Rood B, Vezina G, MacDonald T, Fisher MJ, Cohen B |title=Objective response of multiply recurrent low-grade gliomas to bevacizumab and irinotecan |journal=Pediatr Blood Cancer |volume=52 |issue=7 |pages=791–5 |date=July 2009 |pmid=19165892 |doi=10.1002/pbc.21935 |url=}}</ref> | |||
* | *[[Interferon gamma]] has been proposed for cases of patients with [[Von Recklinghausen's Disease|Von Recklinghausen]] which have intractable or inoperable neurofibromas.<ref name="pmid12692353">{{cite journal |vauthors=Nakayama J, Tanaka T, Arakawa F, Terao H, Shimura H, Ikeda S, Kuroki M |title=Gamma interferon gene transfection efficiently inhibits proliferation of neurofibroma cell lines in vitro |journal=J. Dermatol. |volume=30 |issue=3 |pages=181–8 |date=March 2003 |pmid=12692353 |doi=10.1111/j.1346-8138.2003.tb00369.x |url=}}</ref><ref name="urlscielo.isciii.es" /> | ||
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==References== | ==References== | ||
{{Reflist|2}} | {{Reflist|2}} |
Latest revision as of 16:42, 1 September 2020
Neurofibromatosis type 1 Microchapters |
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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]; Associate Editor(s)-in-Chief: Moises Romo M.D.
Overview
There is no treatment for neurofibromatosis type 1; the mainstay of therapy is supportive care. Therapy for a patient with neurofibromatosis type 1 is aimed at decreasing symptoms and improving quality of life. At every age, different problems may develop, so a specific approach is necessary for the age and presentation of the individual. Supportive therapy for neurofibromatosis type 1 includes pain relief, psychotherapy, and antidepressants.
Medical Therapy
- At every age, different problems may appear, so a specific approach is necessary for the age and presentation of the individual.[1]
- Supportive therapy for neurofibromatosis type 1 includes pain relief, psychotherapy, and antidepressants.[2]
- Stimulant medications should be considered for patients with attention deficitis (ADHD).[3]
- Ketotifen (mast cell blocker that produces its depigmentation) has been proposed to treat some of the symptoms of pruritus, pain, and muscle weakness associated with neurofibromatosis type 1. The dose of ketotifen is 2 to 4 milligrams daily.[4][5]
- Chemotherapy may be necessary for certain tumors.[1] Carboplatin-based chemotherapy combined with vincristine, is the preferred therapy for neurofibromatosis type 1 associated low-grade gliomas.[6]
- Bevacizumab has demonstrated good response on vision testing in cases of refractory optic pathway glioma.[7]
- Interferon gamma has been proposed for cases of patients with Von Recklinghausen which have intractable or inoperable neurofibromas.[8][5]
References
- ↑ 1.0 1.1 . doi:10.1016/B978-0-444-62702-5.00004-4. Check
|doi=
value (help). Missing or empty|title=
(help) - ↑ Listernick, Robert; Charrow, Joel; Greenwald, Mark J.; Esterly, Nancy B. (1989). "Optic gliomas in children with neurofibromatosis type 1". The Journal of Pediatrics. 114 (5): 788–792. doi:10.1016/S0022-3476(89)80137-4. ISSN 0022-3476.
- ↑ Mautner, V.-F.; Kluwe, L.; Friedrich, R. E.; Roehl, A. C.; Bammert, S.; Hogel, J.; Spori, H.; Cooper, D. N.; Kehrer-Sawatzki, H. (2010). "Clinical characterisation of 29 neurofibromatosis type-1 patients with molecularly ascertained 1.4 Mb type-1 NF1 deletions". Journal of Medical Genetics. 47 (9): 623–630. doi:10.1136/jmg.2009.075937. ISSN 0022-2593.
- ↑ Riccardi VM (May 1993). "A controlled multiphase trial of ketotifen to minimize neurofibroma-associated pain and itching". Arch Dermatol. 129 (5): 577–81. PMID 8481017.
- ↑ 5.0 5.1 "scielo.isciii.es" (PDF).
- ↑ Dodgshun AJ, Maixner WJ, Heath JA, Sullivan MJ, Hansford JR (January 2016). "Single agent carboplatin for pediatric low-grade glioma: A retrospective analysis shows equivalent efficacy to multiagent chemotherapy". Int. J. Cancer. 138 (2): 481–8. doi:10.1002/ijc.29711. PMID 26235348.
- ↑ Packer RJ, Jakacki R, Horn M, Rood B, Vezina G, MacDonald T, Fisher MJ, Cohen B (July 2009). "Objective response of multiply recurrent low-grade gliomas to bevacizumab and irinotecan". Pediatr Blood Cancer. 52 (7): 791–5. doi:10.1002/pbc.21935. PMID 19165892.
- ↑ Nakayama J, Tanaka T, Arakawa F, Terao H, Shimura H, Ikeda S, Kuroki M (March 2003). "Gamma interferon gene transfection efficiently inhibits proliferation of neurofibroma cell lines in vitro". J. Dermatol. 30 (3): 181–8. doi:10.1111/j.1346-8138.2003.tb00369.x. PMID 12692353.