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Recombinant macrophage targeted enzyme replacement therapy for Gaucher disease in India.

Publication ,  Journal Article
Nagral, A; Mewawalla, P; Jagadeesh, S; Kabra, M; Phadke, SR; Verma, IC; Puri, RD; Gupta, N; Kishnani, PS; Mistry, PK
Published in: Indian Pediatr
October 2011

OBJECTIVE: Gaucher disease in India has been reported only in a few case reports from India. The aim of the study was to assess the response to enzyme replacement therapy in Indian patients with Gaucher disease. DESIGN: Retrospective analysis of patients receiving CHO-derived recombinant macrophage-targetted glucocorebrosidase. SETTING: Five centers from India with experience in treating lysosomal storage disorders. PATIENTS: The diagnosis of Gaucher disease was confirmed by low glucocerebrosidase levels, though it was first made on splenectomy in 8 and on bone marrow examination in 9 patients. Twenty five of 52 patients diagnosed with Gaucher disease (17 Type I, 8 mild Type III) received treatment for >6 months. Indications for treatment included symptomatic anemia, thrombo-cytopenia, organomegaly, bone disease or mild neurological symptoms leading to impairment of quality of life. Patients with significant neurological involvement were excluded. The drug infusions were given intravenously every 15 days. MAIN OUTCOME MEASURES: Hemoglobin, platelet counts, liver and spleen volumes and growth parameters. RESULTS: 22 of the 25 children who survived were analyzed. After 6 months of treatment, the mean (range) increase in hemoglobin was 1.5 (-3.4 to 6.1) g/dL (P=0.01) and in platelet count was 32 x 10(9)/L (-98.5 x 109 to 145.5 x10(9))/L (P=0.02). The mean (range) increase in weight was 3 kg (-5.6 to 10.5) (P=0.04) and in height was 7.1 cm (0 to 26.5) (P=0.0003). Liver size decreased by a mean (range) of 38.5% (- 5.5 to 86.7) (P=0.0003) and the spleen size by 34.8% (0 to 91.7) (P=0.004). All patients had improvement in bone pains and in 2 patients, neurological symptoms improved with others remaining static. CONCLUSIONS: This is the first reported cohort of patients in India reporting our experience with imiglucerase enzyme replacement therapy for treatment of Gaucher Disease in India.

Duke Scholars

Published In

Indian Pediatr

DOI

EISSN

0974-7559

Publication Date

October 2011

Volume

48

Issue

10

Start / End Page

779 / 784

Location

India

Related Subject Headings

  • Treatment Outcome
  • Retrospective Studies
  • Recombinant Proteins
  • Pediatrics
  • Male
  • Macrophages
  • Infant
  • India
  • Humans
  • Glucosylceramidase
 

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Nagral, A., Mewawalla, P., Jagadeesh, S., Kabra, M., Phadke, S. R., Verma, I. C., … Mistry, P. K. (2011). Recombinant macrophage targeted enzyme replacement therapy for Gaucher disease in India. Indian Pediatr, 48(10), 779–784. https://doi.org/10.1007/s13312-011-0128-4
Nagral, A., P. Mewawalla, S. Jagadeesh, M. Kabra, S. R. Phadke, I. C. Verma, R. D. Puri, N. Gupta, P. S. Kishnani, and P. K. Mistry. “Recombinant macrophage targeted enzyme replacement therapy for Gaucher disease in India.Indian Pediatr 48, no. 10 (October 2011): 779–84. https://doi.org/10.1007/s13312-011-0128-4.
Nagral A, Mewawalla P, Jagadeesh S, Kabra M, Phadke SR, Verma IC, et al. Recombinant macrophage targeted enzyme replacement therapy for Gaucher disease in India. Indian Pediatr. 2011 Oct;48(10):779–84.
Nagral, A., et al. “Recombinant macrophage targeted enzyme replacement therapy for Gaucher disease in India.Indian Pediatr, vol. 48, no. 10, Oct. 2011, pp. 779–84. Pubmed, doi:10.1007/s13312-011-0128-4.
Nagral A, Mewawalla P, Jagadeesh S, Kabra M, Phadke SR, Verma IC, Puri RD, Gupta N, Kishnani PS, Mistry PK. Recombinant macrophage targeted enzyme replacement therapy for Gaucher disease in India. Indian Pediatr. 2011 Oct;48(10):779–784.
Journal cover image

Published In

Indian Pediatr

DOI

EISSN

0974-7559

Publication Date

October 2011

Volume

48

Issue

10

Start / End Page

779 / 784

Location

India

Related Subject Headings

  • Treatment Outcome
  • Retrospective Studies
  • Recombinant Proteins
  • Pediatrics
  • Male
  • Macrophages
  • Infant
  • India
  • Humans
  • Glucosylceramidase