Recombinant Macrophage Targeted Enzyme Replacement Therapy for Gaucher Disease in India.

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dc.contributor.authorNagral, A
dc.contributor.authorMewawalla, P
dc.contributor.authorJagadeesh, S
dc.contributor.authorKabra, M
dc.contributor.authorPhadke, S R
dc.contributor.authorVerma, I C
dc.contributor.authorPuri, R D
dc.contributor.authorGupta, N
dc.contributor.authorKishnani, P S
dc.contributor.authorMistry, P K
dc.date.accessioned2015-12-24T05:35:50Z
dc.date.available2015-12-24T05:35:50Z
dc.date.issued2011-10
dc.description.abstractObjective: 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, thrombocytopenia, 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 109/L (-98.5 x 109 to 145.5 x109) /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.en_US
dc.identifier.citationNagral A, Mewawalla P, Jagadeesh S, Kabra M, Phadke S R, Verma I C, Puri R D, Gupta N, Kishnani P S, Mistry P K. Recombinant Macrophage Targeted Enzyme Replacement Therapy for Gaucher Disease in India. Indian Pediatrics. 2011 October; 48(10): 779-784.en_US
dc.identifier.urihttps://imsear.searo.who.int/handle/123456789/168984
dc.language.isoenen_US
dc.source.urihttps://www.indianpediatrics.net/oct2011/oct-779-784.htmen_US
dc.subjectChildrenen_US
dc.subjectImigluceraseen_US
dc.subjectGaucher diseaseen_US
dc.subjectIndiaen_US
dc.subjectLysosomal storage disorderen_US
dc.subjectTreatmenten_US
dc.titleRecombinant Macrophage Targeted Enzyme Replacement Therapy for Gaucher Disease in India.en_US
dc.typeArticleen_US
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