Achievement of therapeutic goals with low-dose imiglucerase in gaucher disease: A single-center experience

Advances in Hematology

Volumn 2013, Issue , 2013, Pages

  Tukan, Irina ^a   Hadas Halpern, Irith ^a   Altarescu, Gheona ^a   Abrahamov, Ayala ^a   Elstein, Deborah ^a   Zimran, Ari ^a  

^a HEBREW UNIVERSITY   (Israel)

Author keywords

[No Author keywords available]

Indexed keywords

HEMOGLOBIN; IMIGLUCERASE;

ADOLESCENT; ADULT; AGED; ARTICLE; BODY GROWTH; CHILD; ENZYME REPLACEMENT; FEMALE; FEMUR; GAUCHER DISEASE; HUMAN; LIVER WEIGHT; LOW DRUG DOSE; LUMBAR SPINE; MAJOR CLINICAL STUDY; MALE; PRIORITY JOURNAL; PUBERTY; SCHOOL CHILD; SPLEEN; THROMBOCYTE COUNT;

EID: 84888627976     PISSN: 16879104     EISSN: 16879112     Source Type: Journal    
DOI: 10.1155/2013/151506     Document Type: Article

References (15)

1. Zimran A., Elstein D., Lichtman M. A., Kipps T., Seligsohn U., Kaushansky K., Prchal J. T., Lipid storage diseases. Williams Hematology 2010 8th New York, NY, USA McGraw-Hill 1065 1071
2. 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., Grewal R. P., Yu K.-T., Graham O. C., Holder C. A., Howard K. D., Kaneski C. R., Oliver K. L., Riesz S., Verderse C. L., Zirzow G. C., Replacement therapy for inherited enzyme deficiency: macrophage-targeted glucocerebrosidase for Gaucher's disease. The New England Journal of Medicine 1991 324 21 1464 1470 2-s2.0-0025869216
3. 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. Annals of Internal Medicine 1995 122 1 33 39 2-s2.0-0028883136
4. Zimran A., Elstein D., Levy-Lahad E., Zevin S., Hadas-Halpern I., Bar-Ziv Y., Foldes J., Schwartz A. J., Abrahamov A., Replacement therapy with imiglucerase for type 1 Gaucher's disease. The Lancet 1995 345 8963 1479 1480 2-s2.0-0029029221 10.1016/S0140-6736(95)91038-7
5. 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 5 years of treatment: a report from the Gaucher registry. The American Journal of Medicine 2002 113 2 112 119 2-s2.0-0037159549 10.1016/S0002-9343(02)01150-6 (Pubitemid 34804747)
6. Weinreb N. J., Advances in Gaucher disease: therapeutic goals and evaluation and monitoring guidelines. Seminars in Hematology 2004 41 4, supplement 5 1 3 2-s2.0-16644362204
7. Pastores G. M., Weinreb N. J., Aerts H., Andria G., Cox T. M., Giralt M., Grabowski G. A., Mistry P. K., Tylki-Szymańska A., Therapeutic goals in the treatment of Gaucher disease. Seminars in Hematology 2004 41 5, supplement 5 4 14 2-s2.0-4744370348 10.1053/j.seminhematol.2004.07.009 (Pubitemid 39311677)
8. Weinreb N., Taylor J., Cox T., Yee J., vom Dahl S., A benchmark analysis of the achievement of therapeutic goals for type 1 Gaucher disease patients treated with imiglucerase. The American Journal of Hematology 2008 83 12 890 895 2-s2.0-56749130566 10.1002/ajh.21280
9. Elstein D., Abrahamov A., Hadas-Halpern I., Meyer A., Zimran A., Low-dose low-frequency imiglucerase as a starting regimen of enzyme replacement therapy for patients with type I Gaucher disease. Quarterly Journal of Medicine 1998 91 7 483 488 2-s2.0-0031841598 (Pubitemid 28325347)
10. Elstein D., Hadas-Halpern I., Azuri Y., Abrahamov A., Bar-Ziv Y., Zimran A., Accuracy of ultrasonography in assessing spleen and liver size in patients with Gaucher disease: comparison to computed tomographic measurements. Journal of Ultrasound in Medicine 1997 16 3 209 211 2-s2.0-0031104143 (Pubitemid 27391840)
11. Grabowski G. A., Kacena K., Cole J. A., Hollak C. E. M., Zhang L., Yee J., Mistry P. K., Zimran A., Charrow J., Vom Dahl S., Dose-response relationships for enzyme replacement therapy with imiglucerase/alglucerase in patients with Gaucher disease type 1. Genetics in Medicine 2009 11 2 92 100 2-s2.0-62149099925 10.1097/GIM.0b013e31818e2c19
12. Sidransky E., Pastores G. M., Mori M., Dosing enzyme replacement therapy for Gaucher disease: older, but are we wiser? Genetics in Medicine 2009 11 2 90 91 2-s2.0-62149144047 10.1097/GIM.0b013e3181928f6a
13. Andersson H., Kaplan P., Kacena K., Yee J., Eight-year clinical outcomes of long-term enzyme replacement therapy for 884 children with Gaucher disease type 1. Pediatrics 2008 122 6 1182 1190 2-s2.0-49649085254 10.1542/peds.2007- 2144
14. Khan A., Hangartner T., Weinreb N. J., Taylor J. S., Mistry P. K., Risk factors for fractures and avascular osteonecrosis in type 1 Gaucher disease-a study from the international collaborative Gaucher group (ICGG) Gaucher registry. Journal of Bone and Mineral Research 2012 27 8 1839 1848
15. Stein P., Malhotra A., Haims A., Pastores G. M., Mistry P. K., Focal splenic lesions in type I Gaucher disease are associated with poor platelet and splenic response to macrophage-targeted enzyme replacement therapy. Journal of Inherited Metabolic Disease 2010 33 6 769 774 2-s2.0-79952602089 10.1007/s10545-010-9175-6