Difference between revisions of "Erdheim-Chester disease"

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=Langerhans cell histiocytosis (LCH)=
 
=Langerhans cell histiocytosis (LCH)=
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==Cladribine (Leustatin) {{#subobject:da32fb|Regimen=1}}==
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{| class="wikitable" style="float:right; margin-left: 5px;"
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|-
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|[[#toc|back to top]]
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|}
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===Regimen {{#subobject:1ef1c0|Variant=1}}===
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{| border="1" style="text-align:center;" !align="left"
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|'''Study'''
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|[[Levels_of_Evidence#Evidence|'''Evidence''']]
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|-
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|[http://onlinelibrary.wiley.com/doi/10.1002/pbc.22229/abstract Weitzman et al. 2009 (LCH-S-98)]
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|<span
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style="background:#EEEE00;
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padding:3px 6px 3px 6px;
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border-color:black;
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border-width:2px;
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border-style:solid;">Phase II</span>
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|-
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|}
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To be completed
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*[[Cladribine (Leustatin)]]
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===References===
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# Weitzman S, Braier J, Donadieu J, Egeler RM, Grois N, Ladisch S, Pötschger U, Webb D, Whitlock J, Arceci RJ. 2'-Chlorodeoxyadenosine (2-CdA) as salvage therapy for Langerhans cell histiocytosis (LCH). results of the LCH-S-98 protocol of the Histiocyte Society. Pediatr Blood Cancer. 2009 Dec 15;53(7):1271-6. [http://onlinelibrary.wiley.com/doi/10.1002/pbc.22229/abstract link to original article] [http://www.ncbi.nlm.nih.gov/pubmed/19731321 PubMed]
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==Cladribine & Cytarabine {{#subobject:4c5b41|Regimen=1}}==
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{| class="wikitable" style="float:right; margin-left: 5px;"
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|-
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|[[#toc|back to top]]
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|}
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===Regimen {{#subobject:7b85e8|Variant=1}}===
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{| border="1" style="text-align:center;" !align="left"
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|'''Study'''
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|[[Levels_of_Evidence#Evidence|'''Evidence''']]
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|-
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|[http://www.bloodjournal.org/content/126/12/1415.long Donadieu et al. 2015]
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|<span
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style="background:#EEEE00;
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padding:3px 6px 3px 6px;
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border-color:black;
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border-width:2px;
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border-style:solid;">Phase II</span>
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|-
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|}
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*[[Cladribine (Leustatin)]] 9 mg/m2 IV over 2 hours once per day on days 2 to 6, '''should not be administered simultaneously with cytarabine'''
 +
**Children weighing less than 10 kg received a dose of 0.3 mg/kg/day
 +
*[[Cytarabine (Cytosar)]] 500 mg/m2 IV over 2 hours twice per day on days 1 to 5, '''should not be administered simultaneously with cladribine'''
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 +
'''5-week cycle x 2 or more cycles'''
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 +
''Patients with a "good response" proceeded to consolidation therapy:''
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*[[Cladribine (Leustatin)]] 5 mg/m2 IV once per day on days 1 to 3
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'''21-day cycle x 2 cycles, followed by multidrug maintentance therapy:'''
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*[[Vinblastine (Velban)]] 6 mg/m2 IV once every 2 weeks x 6 months
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*[[Prednisolone (Millipred)]] 40 mg/m2 PO once per day on days 1 to 5 of every 2nd week x 6 months
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*[[Mercaptopurine (Purinethol)]] 50 mg/m2 PO once per day x 18 months
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*[[Methotrexate (MTX)]] 20 mg/m2 PO once per week x 18 months
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'''Total of 18 months of maintenance'''
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===References===
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<!-- Preliminary data from this study were presented orally at the 30th meeting of the Histiocyte Society, Washington, DC, 22 October 2013. -->
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# Donadieu J, Bernard F, van Noesel M, Barkaoui M, Bardet O, Mura R, Arico M, Piguet C, Gandemer V, Armari Alla C, Clausen N, Jeziorski E, Lambilliote A, Weitzman S, Henter JI, Van Den Bos C; Salvage Group of the Histiocyte Society. Cladribine and cytarabine in refractory multisystem Langerhans cell histiocytosis: results of an international phase 2 study. Blood. 2015 Sep 17;126(12):1415-23. Epub 2015 Jul 20. [http://www.bloodjournal.org/content/126/12/1415.long link to original article] '''contains verified protocol''' [http://www.ncbi.nlm.nih.gov/pubmed/26194764 PubMed]
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==Etoposide & Prednisone {{#subobject:566a87|Regimen=1}}==
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{| class="wikitable" style="float:right; margin-left: 5px;"
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|-
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|[[#toc|back to top]]
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|}
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===Regimen {{#subobject:#3c6b84|Variant=1}}===
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{| border="1" style="text-align:center;" !align="left"
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|'''Study'''
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|[[Levels_of_Evidence#Evidence|'''Evidence''']]
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|'''Comparator'''
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|-
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|[http://www.jpeds.com/article/S0022-3476(01)07325-5/abstract Gadner et al. 2001 (LCH-I)]
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|<span
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style="background:#00CD00;
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padding:3px 6px 3px 6px;
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border-color:black;
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border-width:2px;
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border-style:solid;">Phase III</span>
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|Vinblastine & Prednisone
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|-
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|}
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To be completed
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*[[Etoposide (Vepesid)]]
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*[[Prednisone (Sterapred)]]
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 +
===References===
 +
# Gadner H, Grois N, Arico M, Broadbent V, Ceci A, Jakobson A, Komp D, Michaelis J, Nicholson S, Pötschger U, Pritchard J, Ladisch S; Histiocyte Society. A randomized trial of treatment for multisystem Langerhans' cell histiocytosis. J Pediatr. 2001 May;138(5):728-34. Erratum in: J Pediatr 2001 Jul;139(1):170. [http://www.jpeds.com/article/S0022-3476(01)07325-5/abstract link to original article] [http://www.ncbi.nlm.nih.gov/pubmed/11343051 PubMed]
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==Etoposide, Vinblastine, Prednisone {{#subobject:5145a8|Regimen=1}}==
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{| class="wikitable" style="float:right; margin-left: 5px;"
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|-
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|[[#toc|back to top]]
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|}
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===Regimen {{#subobject:#7ace0e|Variant=1}}===
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{| border="1" style="text-align:center;" !align="left"
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|'''Study'''
 +
|[[Levels_of_Evidence#Evidence|'''Evidence''']]
 +
|'''Comparator'''
 +
|-
 +
|[http://www.bloodjournal.org/content/111/5/2556.long Gadner et al. 2008 (MCH-II)]
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|<span
 +
style="background:#00CD00;
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padding:3px 6px 3px 6px;
 +
border-color:black;
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border-width:2px;
 +
border-style:solid;">Phase III</span>
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|Vinblastine & Prednisone
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|-
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|}
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To be completed
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*[[Etoposide (Vepesid)]]
 +
*[[Vinblastine (Velban)]]
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*[[Prednisone (Sterapred)]]
 +
 +
===References===
 +
# Gadner H, Grois N, Pötschger U, Minkov M, Aricò M, Braier J, Broadbent V, Donadieu J, Henter JI, McCarter R, Ladisch S; Histiocyte Society. Improved outcome in multisystem Langerhans cell histiocytosis is associated with therapy intensification. Blood. 2008 Mar 1;111(5):2556-62. Epub 2007 Dec 18. [http://www.bloodjournal.org/content/111/5/2556.long link to original article] [http://www.ncbi.nlm.nih.gov/pubmed/18089850 PubMed]
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==Methotrexate, Vinblastine, Prednisone {{#subobject:2d772c|Regimen=1}}==
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{| class="wikitable" style="float:right; margin-left: 5px;"
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|-
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|[[#toc|back to top]]
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|}
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===Regimen {{#subobject:#f569ce|Variant=1}}===
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{| border="1" style="text-align:center;" !align="left"
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|'''Study'''
 +
|[[Levels_of_Evidence#Evidence|'''Evidence''']]
 +
|'''Comparator'''
 +
|-
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|[http://www.bloodjournal.org/content/121/25/5006.long Gadner et al. 2013 (MCH-III)]
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|<span
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style="background:#00CD00;
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padding:3px 6px 3px 6px;
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border-color:black;
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border-width:2px;
 +
border-style:solid;">Phase III</span>
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|Vinblastine & Prednisone
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|-
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|}
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To be completed
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*[[Methotrexate (MTX)]]
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*[[Vinblastine (Velban)]]
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*[[Prednisone (Sterapred)]]
 +
 +
===References===
 +
# Gadner H, Minkov M, Grois N, Pötschger U, Thiem E, Aricò M, Astigarraga I, Braier J, Donadieu J, Henter JI, Janka-Schaub G, McClain KL, Weitzman S, Windebank K, Ladisch S; Histiocyte Society. Therapy prolongation improves outcome in multisystem Langerhans cell histiocytosis. Blood. 2013 Jun 20;121(25):5006-14. Epub 2013 Apr 15. [http://www.bloodjournal.org/content/121/25/5006.long link to original article] [http://www.ncbi.nlm.nih.gov/pubmed/23589673 PubMed]
  
 
==Vemurafenib (Zelboraf) {{#subobject:953978|Regimen=1}}==
 
==Vemurafenib (Zelboraf) {{#subobject:953978|Regimen=1}}==
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===References===
 
===References===
 
# Hyman DM, Puzanov I, Subbiah V, Faris JE, Chau I, Blay JY, Wolf J, Raje NS, Diamond EL, Hollebecque A, Gervais R, Elez-Fernandez ME, Italiano A, Hofheinz RD, Hidalgo M, Chan E, Schuler M, Lasserre SF, Makrutzki M, Sirzen F, Veronese ML, Tabernero J, Baselga J. Vemurafenib in Multiple Nonmelanoma Cancers with BRAF V600 Mutations. N Engl J Med. 2015 Aug 20;373(8):726-736. [http://www.nejm.org/doi/full/10.1056/NEJMoa1502309 link to original article] '''contains protocol''' [http://www.ncbi.nlm.nih.gov/pubmed/26287849 PubMed]
 
# Hyman DM, Puzanov I, Subbiah V, Faris JE, Chau I, Blay JY, Wolf J, Raje NS, Diamond EL, Hollebecque A, Gervais R, Elez-Fernandez ME, Italiano A, Hofheinz RD, Hidalgo M, Chan E, Schuler M, Lasserre SF, Makrutzki M, Sirzen F, Veronese ML, Tabernero J, Baselga J. Vemurafenib in Multiple Nonmelanoma Cancers with BRAF V600 Mutations. N Engl J Med. 2015 Aug 20;373(8):726-736. [http://www.nejm.org/doi/full/10.1056/NEJMoa1502309 link to original article] '''contains protocol''' [http://www.ncbi.nlm.nih.gov/pubmed/26287849 PubMed]
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==Vinblastine & Prednisone {{#subobject:378322|Regimen=1}}==
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{| class="wikitable" style="float:right; margin-left: 5px;"
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|-
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|[[#toc|back to top]]
 +
|}
 +
 +
===Regimen {{#subobject:#bf6a74|Variant=1}}===
 +
{| border="1" style="text-align:center;" !align="left"
 +
|'''Study'''
 +
|[[Levels_of_Evidence#Evidence|'''Evidence''']]
 +
|'''Comparator'''
 +
|-
 +
|[http://www.jpeds.com/article/S0022-3476(01)07325-5/abstract Gadner et al. 2001 (LCH-I)]
 +
|<span
 +
style="background:#00CD00;
 +
padding:3px 6px 3px 6px;
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border-color:black;
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border-width:2px;
 +
border-style:solid;">Phase III</span>
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|Etoposide & Prednisone
 +
|-
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!colspan="4" align="center"|
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|-
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|[http://www.bloodjournal.org/content/111/5/2556.long Gadner et al. 2008 (MCH-II)]
 +
|<span
 +
style="background:#00CD00;
 +
padding:3px 6px 3px 6px;
 +
border-color:black;
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border-width:2px;
 +
border-style:solid;">Phase III</span>
 +
|Etoposide, Vinblastine, Prednisone
 +
|-
 +
!colspan="4" align="center"|
 +
|-
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|[http://www.bloodjournal.org/content/121/25/5006.long Gadner et al. 2013 (MCH-III)]
 +
|<span
 +
style="background:#00CD00;
 +
padding:3px 6px 3px 6px;
 +
border-color:black;
 +
border-width:2px;
 +
border-style:solid;">Phase III</span>
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|Methotrexate, Vinblastine, Prednisone
 +
|-
 +
|}
 +
 +
To be completed
 +
*[[Vinblastine (Velban)]]
 +
*[[Prednisone (Sterapred)]]
 +
 +
===References===
 +
# Gadner H, Grois N, Arico M, Broadbent V, Ceci A, Jakobson A, Komp D, Michaelis J, Nicholson S, Pötschger U, Pritchard J, Ladisch S; Histiocyte Society. A randomized trial of treatment for multisystem Langerhans' cell histiocytosis. J Pediatr. 2001 May;138(5):728-34. Erratum in: J Pediatr 2001 Jul;139(1):170. [http://www.jpeds.com/article/S0022-3476(01)07325-5/abstract link to original article] [http://www.ncbi.nlm.nih.gov/pubmed/11343051 PubMed]
 +
# Gadner H, Grois N, Pötschger U, Minkov M, Aricò M, Braier J, Broadbent V, Donadieu J, Henter JI, McCarter R, Ladisch S; Histiocyte Society. Improved outcome in multisystem Langerhans cell histiocytosis is associated with therapy intensification. Blood. 2008 Mar 1;111(5):2556-62. Epub 2007 Dec 18. [http://www.bloodjournal.org/content/111/5/2556.long link to original article] [http://www.ncbi.nlm.nih.gov/pubmed/18089850 PubMed]
 +
# Gadner H, Minkov M, Grois N, Pötschger U, Thiem E, Aricò M, Astigarraga I, Braier J, Donadieu J, Henter JI, Janka-Schaub G, McClain KL, Weitzman S, Windebank K, Ladisch S; Histiocyte Society. Therapy prolongation improves outcome in multisystem Langerhans cell histiocytosis. Blood. 2013 Jun 20;121(25):5006-14. Epub 2013 Apr 15. [http://www.bloodjournal.org/content/121/25/5006.long link to original article] [http://www.ncbi.nlm.nih.gov/pubmed/23589673 PubMed]

Revision as of 15:02, 24 October 2015

Use of this site is subject to you reading and agreeing with the terms set forth in the disclaimer.

Is there a regimen missing from this list? Would you like to share a different dosage/schedule or an additional reference for a regimen? Have you noticed an error? Do you have an idea that will help the site grow to better meet your needs and the needs of many others? You are invited to contribute to the site.

3 regimens on this page
4 variants on this page


Erdheim-Chester disease (ECD)

Sirolimus & Prednisone

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Regimen

Study Evidence
Gianfreda et al. 2015 Pilot, <20 patients in this arm
  • Sirolimus (Rapamune) 2 mg PO once per day, titrated to achieve a blood level of 8 to 12 ng/mL
  • Prednisone (Sterapred) as follows:
    • Month 1: 0.75 mg/kg/day PO
    • Month 2: 0.5 mg/k/day PO
    • Months 3 & 4: 0.25 mg/kg/day PO
    • Months 5 & 6: 0.125 mg/kg/day PO
    • Month 7 onwards: tapered to a dose of 2.5 to 5 mg PO once per day

Given for up to 2 years for responders and stable disease, with optional extension beyond 2 years

References

  1. Gianfreda D, Nicastro M, Galetti M, Alberici F, Corradi D, Becchi G, Baldari G, De Filippo M, Ferretti S, Moroni G, Foti R, Di Gangi M, Jeannin G, Saffroy R, Emile JF, Buzio C, Vaglio A. Sirolimus plus prednisone for Erdheim-Chester disease: an open-label trial. Blood. 2015 Sep 3;126(10):1163-71. Epub 2015 Jun 3. link to original article contains verified protocol PubMed

Vemurafenib (Zelboraf)

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Regimen #1

Study Evidence
Hyman et al. 2015 Phase II, <20 patients in this arm

This was part of a basket trial, all patients had BRAF V600E mutations.

Duration of treatment not specified

Regimen #2

Study Evidence
Haroche et al. 2014 Pilot, <20 patients

Patients enrolled all had BRAF V600E mutation. This is the dose after de-escalation after the first four patients had excess adverse events.

Given until progression of disease or unacceptable toxicity

References

  1. Haroche J, Cohen-Aubart F, Emile JF, Maksud P, Drier A, Tolédano D, Barete S, Charlotte F, Cluzel P, Donadieu J, Benameur N, Grenier PA, Besnard S, Ory JP, Lifermann F, Idbaih A, Granel B, Graffin B, Hervier B, Arnaud L, Amoura Z. Reproducible and Sustained Efficacy of Targeted Therapy With Vemurafenib in Patients With BRAFV600E-Mutated Erdheim-Chester Disease. J Clin Oncol. 2015 Feb 10;33(5):411-8. Epub 2014 Nov 24. link to original article contains verified protocol PubMed
  2. Hyman DM, Puzanov I, Subbiah V, Faris JE, Chau I, Blay JY, Wolf J, Raje NS, Diamond EL, Hollebecque A, Gervais R, Elez-Fernandez ME, Italiano A, Hofheinz RD, Hidalgo M, Chan E, Schuler M, Lasserre SF, Makrutzki M, Sirzen F, Veronese ML, Tabernero J, Baselga J. Vemurafenib in Multiple Nonmelanoma Cancers with BRAF V600 Mutations. N Engl J Med. 2015 Aug 20;373(8):726-736. link to original article contains protocol PubMed

Langerhans cell histiocytosis (LCH)

Cladribine (Leustatin)

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Regimen

Study Evidence
Weitzman et al. 2009 (LCH-S-98) Phase II

To be completed

References

  1. Weitzman S, Braier J, Donadieu J, Egeler RM, Grois N, Ladisch S, Pötschger U, Webb D, Whitlock J, Arceci RJ. 2'-Chlorodeoxyadenosine (2-CdA) as salvage therapy for Langerhans cell histiocytosis (LCH). results of the LCH-S-98 protocol of the Histiocyte Society. Pediatr Blood Cancer. 2009 Dec 15;53(7):1271-6. link to original article PubMed

Cladribine & Cytarabine

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Regimen

Study Evidence
Donadieu et al. 2015 Phase II
  • Cladribine (Leustatin) 9 mg/m2 IV over 2 hours once per day on days 2 to 6, should not be administered simultaneously with cytarabine
    • Children weighing less than 10 kg received a dose of 0.3 mg/kg/day
  • Cytarabine (Cytosar) 500 mg/m2 IV over 2 hours twice per day on days 1 to 5, should not be administered simultaneously with cladribine

5-week cycle x 2 or more cycles

Patients with a "good response" proceeded to consolidation therapy:

21-day cycle x 2 cycles, followed by multidrug maintentance therapy:

Total of 18 months of maintenance

References

  1. Donadieu J, Bernard F, van Noesel M, Barkaoui M, Bardet O, Mura R, Arico M, Piguet C, Gandemer V, Armari Alla C, Clausen N, Jeziorski E, Lambilliote A, Weitzman S, Henter JI, Van Den Bos C; Salvage Group of the Histiocyte Society. Cladribine and cytarabine in refractory multisystem Langerhans cell histiocytosis: results of an international phase 2 study. Blood. 2015 Sep 17;126(12):1415-23. Epub 2015 Jul 20. link to original article contains verified protocol PubMed

Etoposide & Prednisone

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Regimen

Study Evidence Comparator
Gadner et al. 2001 (LCH-I) Phase III Vinblastine & Prednisone

To be completed

References

  1. Gadner H, Grois N, Arico M, Broadbent V, Ceci A, Jakobson A, Komp D, Michaelis J, Nicholson S, Pötschger U, Pritchard J, Ladisch S; Histiocyte Society. A randomized trial of treatment for multisystem Langerhans' cell histiocytosis. J Pediatr. 2001 May;138(5):728-34. Erratum in: J Pediatr 2001 Jul;139(1):170. link to original article PubMed

Etoposide, Vinblastine, Prednisone

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Regimen

Study Evidence Comparator
Gadner et al. 2008 (MCH-II) Phase III Vinblastine & Prednisone

To be completed

References

  1. Gadner H, Grois N, Pötschger U, Minkov M, Aricò M, Braier J, Broadbent V, Donadieu J, Henter JI, McCarter R, Ladisch S; Histiocyte Society. Improved outcome in multisystem Langerhans cell histiocytosis is associated with therapy intensification. Blood. 2008 Mar 1;111(5):2556-62. Epub 2007 Dec 18. link to original article PubMed

Methotrexate, Vinblastine, Prednisone

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Regimen

Study Evidence Comparator
Gadner et al. 2013 (MCH-III) Phase III Vinblastine & Prednisone

To be completed

References

  1. Gadner H, Minkov M, Grois N, Pötschger U, Thiem E, Aricò M, Astigarraga I, Braier J, Donadieu J, Henter JI, Janka-Schaub G, McClain KL, Weitzman S, Windebank K, Ladisch S; Histiocyte Society. Therapy prolongation improves outcome in multisystem Langerhans cell histiocytosis. Blood. 2013 Jun 20;121(25):5006-14. Epub 2013 Apr 15. link to original article PubMed

Vemurafenib (Zelboraf)

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Regimen

Study Evidence
Hyman et al. 2015 Phase II, <20 patients in this arm

This was part of a basket trial, all patients had BRAF V600E mutations.

Duration of treatment not specified

References

  1. Hyman DM, Puzanov I, Subbiah V, Faris JE, Chau I, Blay JY, Wolf J, Raje NS, Diamond EL, Hollebecque A, Gervais R, Elez-Fernandez ME, Italiano A, Hofheinz RD, Hidalgo M, Chan E, Schuler M, Lasserre SF, Makrutzki M, Sirzen F, Veronese ML, Tabernero J, Baselga J. Vemurafenib in Multiple Nonmelanoma Cancers with BRAF V600 Mutations. N Engl J Med. 2015 Aug 20;373(8):726-736. link to original article contains protocol PubMed

Vinblastine & Prednisone

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Regimen

Study Evidence Comparator
Gadner et al. 2001 (LCH-I) Phase III Etoposide & Prednisone
Gadner et al. 2008 (MCH-II) Phase III Etoposide, Vinblastine, Prednisone
Gadner et al. 2013 (MCH-III) Phase III Methotrexate, Vinblastine, Prednisone

To be completed

References

  1. Gadner H, Grois N, Arico M, Broadbent V, Ceci A, Jakobson A, Komp D, Michaelis J, Nicholson S, Pötschger U, Pritchard J, Ladisch S; Histiocyte Society. A randomized trial of treatment for multisystem Langerhans' cell histiocytosis. J Pediatr. 2001 May;138(5):728-34. Erratum in: J Pediatr 2001 Jul;139(1):170. link to original article PubMed
  2. Gadner H, Grois N, Pötschger U, Minkov M, Aricò M, Braier J, Broadbent V, Donadieu J, Henter JI, McCarter R, Ladisch S; Histiocyte Society. Improved outcome in multisystem Langerhans cell histiocytosis is associated with therapy intensification. Blood. 2008 Mar 1;111(5):2556-62. Epub 2007 Dec 18. link to original article PubMed
  3. Gadner H, Minkov M, Grois N, Pötschger U, Thiem E, Aricò M, Astigarraga I, Braier J, Donadieu J, Henter JI, Janka-Schaub G, McClain KL, Weitzman S, Windebank K, Ladisch S; Histiocyte Society. Therapy prolongation improves outcome in multisystem Langerhans cell histiocytosis. Blood. 2013 Jun 20;121(25):5006-14. Epub 2013 Apr 15. link to original article PubMed
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