Evaluation of miglustat as maintenance therapy after enzyme therapy in adults with stable type 1 Gaucher disease: A prospective, open-label non-inferiority study

Orphanet Journal of Rare Diseases

Volumn 7, Issue 1, 2012, Pages

  Cox, Timothy M ^a   Amato, Dominick ^b   Hollak, Carla Em ^c   Luzy, Cecile ^d   Silkey, Mariabeth ^d   Giorgino, Ruben ^d   Steiner, Robert D ^e  

^a UNIVERSITY OF CAMBRIDGE   (United Kingdom)

^b UNIVERSITY OF TORONTO   (Canada)

^c ACADEMIC MEDICAL CENTER   (Netherlands)

^d ACTELION PHARMACEUTICALS LTD   (Switzerland)

^e OREGON HEALTH AND SCIENCE UNIVERSITY   (United States)

Author keywords

Efficacy; Enzyme therapy; Gaucher disease; Maintenance; Miglustat; Safety

Indexed keywords

HEMOGLOBIN; MIGLUSTAT;

ABDOMINAL DISCOMFORT; ABDOMINAL DISTENSION; ADULT; ARTHRALGIA; ARTICLE; BODY WEIGHT; CEREBELLUM DISEASE; CLINICAL ARTICLE; DIARRHEA; DIZZINESS; DRUG DOSE REDUCTION; DRUG INDUCED HEADACHE; DRUG SAFETY; DRUG TOLERABILITY; DRUG WITHDRAWAL; ENZYME THERAPY; FATIGUE; FEMALE; FLATULENCE; GASTROINTESTINAL SYMPTOM; GAUCHER DISEASE; HUMAN; HYPERREFLEXIA; HYPESTHESIA; LIVER WEIGHT; MAINTENANCE THERAPY; MALE; MULTICENTER STUDY; PARESTHESIA; PHASE 3 CLINICAL TRIAL; PROSPECTIVE STUDY; SIDE EFFECT; SPLEEN; THROMBOCYTE COUNT; TREATMENT DURATION; TREMOR;

1-DEOXYNOJIRIMYCIN; ADULT; ENZYME INHIBITORS; ENZYME THERAPY; FEMALE; GAUCHER DISEASE; HUMANS; MALE; MIDDLE AGED; PROSPECTIVE STUDIES; TREATMENT OUTCOME;

EID: 84871603938     PISSN: None     EISSN: 17501172     Source Type: Journal    
DOI: 10.1186/1750-1172-7-102     Document Type: Article

References (38)

1. Gaucher disease. Beutler E, Grabowski GA, The metabolic and molecular basis of inherited disease New York: McGraw-Hill, Scriver CR, Beaudet AL, Sly WS, Valle D, 8 1995 3635 3668
2. The Gaucher registry: demographics and disease characteristics of 1698 patients with Gaucher disease. Charrow J, Andersson HC, Kaplan P, Kolodny EH, Mistry P, Pastores G, Rosenbloom BE, Scott CR, Wappner RS, Weinreb NJ, Zimran A, Arch Intern Med 2000 160 2835 2843 10.1001/archinte.160.18.2835 11025794
3. Gaucher's disease: clinical features and natural history. Cox TM, Schofield JP, Baillieres Clin Haematol 1997 10 657 689 10.1016/S0950-3536(97) 80033-9 9497857 (Pubitemid 28022202)
4. Peripheral neuropathy in adult type 1 Gaucher disease: a 2-year prospective observational study. Biegstraaten M, Mengel E, Marodi L, Petakov M, Niederau C, Giraldo P, Hughes D, Mrsic M, Mehta A, Hollak CE, van Schaik IN, Brain 2010 133 2909 2919 10.1093/brain/awq198 20693542
5. Gaucher disease: complexity in a simple disorder. Sidransky E, Mol Genet Metab 2004 83 6 15 10.1016/j.ymgme.2004.08.015 15464415 (Pubitemid 39311276)
6. Phase 1/2 and extension study of velaglucerase alfa replacement therapy in adults with type 1 Gaucher disease: 48-month experience. Zimran A, Altarescu G, Philips M, Attias D, Jmoudiak M, Deeb M, Wang N, Bhirangi K, Cohn GM, Elstein D, Blood 2010 115 4651 4656 10.1182/blood-2010-02-268649 20299511
7. Review of the safety and efficacy of imiglucerase treatment of Gaucher disease. Elstein D, Zimran A, Biologics 2009 3 407 417 19774208
8. The long-term international safety experience of imiglucerase therapy for Gaucher disease. Starzyk K, Richards S, Yee J, Smith SE, Kingma W, Mol Genet Metab 2007 90 157 163 10.1016/j.ymgme.2006.09.003 17079176 (Pubitemid 46108612)
9. Significance of immune response to enzyme-replacement therapy for patients with a lysosomal storage disorder. Brooks DA, Kakavanos R, Hopwood JJ, Trends Mol Med 2003 9 450 453 10.1016/j.molmed.2003.08.004 14557058 (Pubitemid 37309647)
10. Evaluation of Spanish Gaucher disease patients after a 6-month imiglucerase shortage. Giraldo P, Irun P, Alfonso P, Dalmau J, Fernandez-Galan MA, Figueredo A, Hernandez-Rivas JM, Julia A, Luno E, Marin-Jimenez F, et al. Blood Cells Mol Dis 2011 46 115 118 10.1016/j.bcmd.2010.09.005 20934891
11. Actelion: zavesca® (miglustat capsules) prescribing information 2010 Available at: http://www.zavesca.com/pdfs/zavesca-pi.pdf. Accessed 9 June, 2011 18339196
12. Novel oral treatment of Gaucher's disease with N-butyldeoxynojirimycin (OGT 918) to decrease substrate biosynthesis. Cox T, Lachmann R, Hollak C, Aerts J, van Weely S, Hrebicek M, Platt F, Butters T, Dwek R, Moyses C, et al. Lancet 2000 355 1481 1485 10.1016/S0140-6736(00)02161-9 10801168 (Pubitemid 30237353)
13. Sustained therapeutic effects of oral miglustat (Zavesca, N-butyldeoxynojirimycin, OGT 918) in type I Gaucher disease. Elstein D, Hollak C, Aerts JM, van Weely S, Maas M, Cox TM, Lachmann RH, Hrebicek M, Platt FM, Butters TD, et al. J Inherit Metab Dis 2004 27 757 766 15505381 (Pubitemid 39433603)
14. Low-dose N-butyldeoxynojirimycin (OGT 918) for type I Gaucher disease. Heitner R, Elstein D, Aerts J, Weely S, Zimran A, Blood Cells Mol Dis 2002 28 127 133 10.1006/bcmd.2002.0497 12064906 (Pubitemid 34743745)
15. An open-label, noncomparative study of miglustat in type I Gaucher disease: efficacy and tolerability over 24 months of treatment. Pastores GM, Barnett NL, Kolodny EH, Clin Ther 2005 27 1215 1227 10.1016/j.clinthera.2005.08. 004 16199246 (Pubitemid 41393915)
16. Real-world clinical experience with long-term miglustat maintenance therapy in type 1 Gaucher disease: the ZAGAL project. Giraldo P, Alfonso P, Atutxa K, Fernandez-Galan MA, Barez A, Franco R, Alonso D, Martin A, Latre P, Pocovi M, Haematologica 2009 94 1771 1775 10.3324/haematol.2009.008078 19608672
17. Short-term effect of miglustat in every day clinical use in treatment-naive or previously treated patients with type 1 Gaucher's disease. Giraldo P, Latre P, Alfonso P, Acedo A, Alonso D, Barez A, Corrales A, Franco R, Roldan V, Serrano S, Pocovi M, Haematologica 2006 91 703 706 16627252 (Pubitemid 43799475)
18. Effect of miglustat on bone disease in adults with type 1 Gaucher disease: a pooled analysis of three multinational, open-label studies. Pastores GM, Elstein D, Hrebicek M, Zimran A, Clin Ther 2007 29 1645 1654 10.1016/j.clinthera.2007.08.006 17919546 (Pubitemid 47494543)
19. Oral maintenance clinical trial with miglustat for type I Gaucher disease: switch from or combination with intravenous enzyme replacement. Elstein D, Dweck A, Attias D, Hadas-Halpern I, Zevin S, Altarescu G, Aerts JF, van Weely S, Zimran A, Blood 2007 110 2296 2301 10.1182/blood-2007-02-075960 17609429 (Pubitemid 47523147)
20. Quantification of bone involvement in Gaucher disease: MR imaging bone marrow burden score as an alternative to Dixon quantitative chemical shift MR imaging-initial experience. Maas M, van Kuijk C, Stoker J, Hollak CE, Akkerman EM, Aerts JF, den Heeten GJ, Radiology 2003 229 554 561 10.1148/radiol. 2292020296 14526090 (Pubitemid 37310846)
21. Therapeutic goals in the treatment of Gaucher disease. Pastores GM, Weinreb NJ, Aerts H, Andria G, Cox TM, Giralt M, Grabowski GA, Mistry PK, Tylki-Szymanska A, Semin Hematol 2004 41 4 14 15468045 (Pubitemid 39311677)
22. Psychometric evaluation of a new sensory scale in immune-mediated polyneuropathies. Inflammatory Neuropathy Cause and Treatment (INCAT) Group. Merkies IS, Schmitz PI, Van der Meche FG, van Doorn PA, Neurology 2000 54 943 949 10.1212/WNL.54.4.943 10690990 (Pubitemid 30111356)
23. Interobserver agreement in the assessment of muscle strength and functional abilities in Guillain-Barre syndrome. Kleyweg RP, van der Meche FG, Schmitz PI, Muscle Nerve 1991 14 1103 1109 10.1002/mus.880141111 1745285
24. AAEM minimonograph #34: polyneuropathy: classification by nerve conduction studies and electromyography. Donofrio PD, Albers JW, Muscle Nerve 1990 13 889 903 10.1002/mus.880131002 2172810 (Pubitemid 20341507)
25. The memory enhancing effects of a Ginkgo biloba/Panax ginseng combination in healthy middle-aged volunteers. Wesnes KA, Ward T, McGinty A, Petrini O, Psychopharmacology 2000 152 353 361 10.1007/s002130000533 11140327
26. The value of assessing cognitive function in drug development. Wesnes KA, Dialogues Clin Neurosci 2000 2 183 202 22033754
27. The use of analogue scales in rating self-rated feelings. Bond A, Lader M, Br J Med Psychol 1974 47 211 218 10.1111/j.2044-8341.1974.tb02285.x
28. The clinical course of treated and untreated Gaucher disease. A study of 45 patients. Beutler E, Demina A, Laubscher K, Garver P, Gelbart T, Balicki D, Vaughan L, Blood Cells Mol Dis 1995 21 86 108 10.1006/bcmd.1995.0012 8846048
29. Low frequency maintenance therapy with imiglucerase in adult type I Gaucher disease: a prospective randomized controlled trial. de Fost M, Aerts JM, Groener JE, Maas M, Akkerman EM, Wiersma MG, Hollak CE, Haematologica 2007 92 215 221 10.3324/haematol.10635 17296571
30. Cessation of enzyme replacement therapy in Gaucher disease. Grinzaid KA, Geller E, Hanna SL, Elsas LJ 2nd, Genet Med 2002 4 427 433 10.1097/00125817- 200211000-00005 12509713 (Pubitemid 44698535)
31. Rebound hepatosplenomegaly in type 1 Gaucher disease. Toth J, Erdos M, Marodi L, Eur J Haematol 2003 70 125 128 10.1034/j.1600-0609.2003.00010.x 12581195 (Pubitemid 36269560)
32. Force majeure: therapeutic measures in response to restricted supply of imiglucerase (Cerezyme) for patients with Gaucher disease. Hollak CE, Vom Dahl S, Aerts JM, Belmatoug N, Bembi B, Cohen Y, Collin-Histed T, Deegan P, Van Dussen L, Giraldo P, et al. Blood Cells Mol Dis 2010 44 41 47 10.1016/j.bcmd.2009.09.006 19804996
33. Nonprecipitous changes upon withdrawal from imiglucerase for Gaucher disease because of a shortage in supply. Zimran A, Altarescu G, Elstein D, Blood Cells Mol Dis 2011 46 111 114 10.1016/j.bcmd.2010.05.001 20542712
34. Changes of bone metabolism in seven patients with Gaucher disease treated consecutively with imiglucerase and miglustat. Mikosch P, Reed M, Baker R, Holloway B, Berger L, Mehta AB, Hughes DA, Calcif Tissue Int 2008 83 43 54 10.1007/s00223-008-9143-4 18553043
35. Guidance on the use of miglustat for treating patients with type 1 Gaucher disease. Weinreb NJ, Barranger JA, Charrow J, Grabowski GA, Mankin HJ, Mistry P, Am J Hematol 2005 80 223 229 10.1002/ajh.20504 16247743 (Pubitemid 41579546)
36. Miglustat (Zavesca) in type 1 Gaucher disease: 5-year results of a post-authorisation safety surveillance programme. Hollak CE, Hughes D, van Schaik IN, Schwierin B, Bembi B, Pharmacoepidemiol Drug Saf 2009 18 770 777 10.1002/pds.1779 19507165
37. Gastrointestinal disturbances and their management in miglustat-treated patients. Belmatoug N, Burlina A, Giraldo P, Hendriksz CJ, Kuter DJ, Mengel E, Pastores GM, J Inherit Metab Dis 2011 34 991 1001 10.1007/s10545-011-9368-7 21779792
38. Dietary modifications in patients receiving miglustat. Champion H, Ramaswami U, Imrie J, Lachmann RH, Gallagher J, Cox TM, Wraith JE, J Inherit Metab Dis 2010 Epub ahead of print 10.1007/s10545-008-0923-9