Significant and continuous improvement in bone mineral density among type 1 Gaucher disease patients treated with velaglucerase alfa: 69-month experience, including dose reduction

Blood Cells, Molecules, and Diseases

Volumn 47, Issue 1, 2011, Pages 56-61

  Elstein, Deborah ^a   Foldes, A Joseph ^b   Zahrieh, David ^c   Cohn, Gabriel M ^c   Djordjevic, Maja ^d   Brutaru, Costin ^e   Zimran, Ari ^a  

^a SHAARE ZEDEK MEDICAL CENTER   (Israel)

^b HADASSAH HEBREW UNIVERSITY MEDICAL CENTER   (Israel)

^c SHIRE   (United States)

^d Mother and Child Health Care Institute   (Serbia)

^e Sf Ioan Clinical Hospital   (Romania)

Author keywords

Bone mineral density; Enzyme replacement therapy; Gaucher disease; Velaglucerase alfa

Indexed keywords

BISPHOSPHONIC ACID DERIVATIVE; VELAGLUCERASE ALFA;

ADULT; AGED; ARTICLE; BONE DENSITY; CLINICAL ARTICLE; DRUG DOSE REDUCTION; DRUG EFFICACY; DRUG SAFETY; FEMALE; FEMUR NECK; GAUCHER DISEASE; GAUCHER DISEASE TYPE 1; HUMAN; LUMBAR SPINE; MALE; OSTEOPENIA; OSTEOPOROSIS; PHASE 1 CLINICAL TRIAL; PHASE 2 CLINICAL TRIAL; PRIORITY JOURNAL; SCORING SYSTEM;

ADOLESCENT; ADULT; BONE DENSITY; BONE DENSITY CONSERVATION AGENTS; DIPHOSPHONATES; ENZYME REPLACEMENT THERAPY; FEMALE; GAUCHER DISEASE; GLUCOSYLCERAMIDASE; HUMANS; MALE; MIDDLE AGED; TREATMENT OUTCOME; YOUNG ADULT;

EID: 79956319911     PISSN: 10799796     EISSN: 10960961     Source Type: Journal    
DOI: 10.1016/j.bcmd.2011.04.005     Document Type: Article

References (23)

1. Zimran A., Elstein D. Lipid storage diseases. Williams Hematology 2010, 1065-1071. McGraw-Hill, New York. 8th edition. M.A. Lichtman, T. Kipps, U. Seligsohn, K. Kaushansky, J.T. Prchal (Eds.).
2. Elstein D., Itzchaki M., Mankin H.J. Skeletal involvement in Gaucher's disease. Baillieres Clin. Haematol. 1997, 10:793-816.
3. E. Beutler and G.A. Grabowski, Gaucher disease, Editors: C.R. Scriver, A.L. Beaudet, W.S. Sly, D. Valle, Associate Editors: B. Childs, K.W. Kinzler, B. Vogelstein, The Metabolic and Molecular Bases of Inherited Disease, 8th edition, McGraw-Hill, New York, 2001, Chapter 146 pp. 3635-3668. ISBNs 0-070913035-6; 07016319-X (Vol 1); 0-07016320-3 (Vol 2); 0-07016321-X (Vol 3); 0-07016322-X (Vol 4).
4. Damiano A.M., Pastores G.M., Ware J.E. The health-related quality of life of adults with Gaucher's disease receiving enzyme replacement therapy: results from a retrospective study. Qual. Life Res. 1998, 7:373-386.
5. Giraldo P., Solano V., Perez-Calvo J.I., Giralt M., Rubio-Felix D. Quality of life related to type 1 Gaucher disease: Spanish experience. Qual. Life Res. 2005, 14:453-462.
6. Pastores G.M. Velaglucerase alfa, a human recombinant glucocerebrosidase enzyme replacement therapy for type 1 Gaucher disease. Curr. Opin. Investig. Drugs 2010, 11:472-478.
7. Zimran A., Loveday K., Fratazzi C., Elstein D. A pharmacokinetic analysis of a novel enzyme replacement therapy with Gene-Activated human glucocerebrosidase (GA-GCB) in patients with type 1 Gaucher disease. Blood Cells Mol. Dis. 2007, 39:115-118.
8. Brumshtein B., Salinas P., Peterson B., Chan V., Silman I., Sussman J.L., Savickas P.J., Robinson G.S., Futerman A.H. Characterization of gene-activated human acid-beta-glucosidase: crystal structure, glycan composition, and internalization into macrophages. Glycobiology 2010, 20:24-32.
9. Zimran A., Altarescu G., Philips M., Attias D., Jmoudiak M., Deeb M., Wang N., Bhirangi K., Cohn G.M., Elstein D. Phase 1/2 and extension study of velaglucerase alfa replacement therapy in adults with type 1 Gaucher disease: 48-month experience. Blood 2010, 115:4651-4656.
10. Pastores G.M., Weinreb N.J., Aerts H., Andria G., Cox T.M., Giralt M., Grabowski G.A., Mistry P.K., Tylki-Szymanska A. Therapeutic goals in the treatment of Gaucher disease. Semin. Hematol. 2004, 41:4-14.
11. Pastores G.M., Wallenstein S., Desnick R.J., Luckey M.M. Bone density in Type 1 Gaucher disease. J. Bone Miner. Res. 1996, 11:1801-1807.
12. Laird N.M., Ware J.H. Random-effects models for longitudinal data. Biometrics 1982, 38:963-974.
13. World Health Organization Assessment of fracture risk and its application to screening for postmenopausal osteoporosis: report of a WHO study group. WHO Technical Report Series, Report No. 843 1994, WHO, Geneva, Switzerland.
14. Barton N.W., Brady R.O., Dambrosia J.M., Di Bisceglie A.M., Doppelt S.H., Hill S.C., Mankin H.J., Murray G.J., Parker R.I., Argoff C.E., et al. Replacement therapy for inherited enzyme deficiency-macrophage-targeted glucocerebrosidase for Gaucher's disease. N. Engl. J. Med. 1991, 324:1464-1470.
15. Grabowski G.A., Barton N.W., Pastores G., Dambrosia J.M., Banerjee T.K., McKee M.A., Parker C., Schiffmann R., Hill S.C., Brady R.O. Enzyme therapy in type 1 Gaucher disease: comparative efficacy of mannose-terminated glucocerebrosidase from natural and recombinant sources. Ann. Intern. Med. 1995, 122:33-39.
16. Rosenthal D.I., Doppelt S.H., Mankin H.J., Dambrosia J.M., Xavier R.J., McKusick K.A., Rosen B.R., Baker J., Niklason L.T., Hill S.C., et al. Enzyme replacement therapy for Gaucher disease: skeletal responses to macrophage-targeted glucocerebrosidase. Pediatrics 1995, 96:629-637.
17. Wenstrup R.J., Kacena K.A., Kaplan P., Pastores G.M., Prakash-Cheng A., Zimran A., Hangartner T.N. Effect of enzyme replacement therapy with imiglucerase on BMD in type 1 Gaucher disease. J. Bone Miner. Res. 2007, 22:119-126.
18. Weinreb N.J., Charrow J., Andersson H.C., Kaplan P., Kolodny E.H., Mistry P., Pastores G., Rosenbloom B.E., Scott C.R., Wappner R.S., Zimran A. Effectiveness of enzyme replacement therapy in 1028 patients with type 1 Gaucher disease after 2 to 5years of treatment: a report from the Gaucher Registry. Am. J. Med. 2002, 113:112-119.
19. Charrow J., Dulisse B., Grabowski G.A., Weinreb N.J. The effect of enzyme replacement therapy on bone crisis and bone pain in patients with type 1 Gaucher disease. Clin. Genet. 2007, 71:205-211.
20. Piran S., Amato D. Gaucher disease: a systematic review and meta-analysis of bone complications and their response to treatment. J. Inherit. Metab. Dis. 2010, 33:271-279.
21. Mistry P.K., Weinreb N.J., Kaplan P., Cole J.A., Gwosdow A.R., Hangartner T. Osteopenia in Gaucher disease develops early in life: response to imiglucerase enzyme therapy in children, adolescents and adults. Blood Cells Mol. Dis. 2011, 46:66-72.
22. Pastores G.M., Elstein D., Hrebicek M., Zimran A. Effect of miglustat on bone disease in adults with type 1 Gaucher disease: a pooled analysis of three multinational, open-label studies. Clin. Ther. 2007, 29:1645-1654.
23. Lukina E., Watman N., Arreguin E.A., Dragosky M., Iastrebner M., Rosenbaum H., Phillips M., Pastores G.M., Kamath R.S., Rosenthal D.I., Kaper M., Singh T., Puga A.C., Peterschmitt M.J. Improvement in hematological, visceral, and skeletal manifestations of Gaucher disease type 1 with oral eliglustat tartrate (Genz-112638) treatment: 2-year results of a phase 2 study. Blood 2010, 116:4095-4098.