Clinical Consequences of Interrupting Enzyme Replacement Therapy in Children with Type 1 Gaucher Disease

Journal of Pediatrics

Volumn 151, Issue 2, 2007, Pages 197-201

  Drelichman, Guillermo ^a   Ponce, Elvira ^a   Basack, Nora ^a   Freigeiro, Daniel ^a   Aversa, Luis ^a   Graciela, Elena ^a   Kohan, Regina ^a  

^a HOSPITAL DE NIÑOS RICARDO GUTIÉRREZ   (Argentina)

Author keywords

[No Author keywords available]

Indexed keywords

ALGLUCERASE; IMIGLUCERASE;

ANEMIA; ARTICLE; BONE DISEASE; CHILD GROWTH; CLINICAL ARTICLE; CLINICAL ASSESSMENT; CLINICAL EXAMINATION; ENZYME REPLACEMENT; FEMALE; GAUCHER DISEASE; GROWTH RETARDATION; HEMATOLOGICAL PARAMETERS; HEMOGLOBIN BLOOD LEVEL; HUMAN; MALE; OUTCOME ASSESSMENT; PRIORITY JOURNAL; SPLENOMEGALY; THROMBOCYTE COUNT; THROMBOCYTOPENIA; TREATMENT OUTCOME;

ADOLESCENT; CHILD; CHILD, PRESCHOOL; COHORT STUDIES; DOSE-RESPONSE RELATIONSHIP, DRUG; DRUG ADMINISTRATION SCHEDULE; ENZYMES; FEMALE; FEMUR HEAD NECROSIS; FOLLOW-UP STUDIES; GAUCHER DISEASE; GLUCOSYLCERAMIDASE; HEMOGLOBINS; HEPATOMEGALY; HUMANS; INFANT; MALE; RETROSPECTIVE STUDIES; RISK ASSESSMENT; THROMBOCYTOPENIA; TREATMENT REFUSAL;

EID: 34447507878     PISSN: 00223476     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.jpeds.2007.02.057     Document Type: Article

References (17)

1. Beutler E., and Grabowski G. Gaucher disease. In: Scriver C., Beaudet A., Sly W., Valle D., et al. (Eds). The metabolic and molecular bases of inherited disease (2001), McGraw Hill, New York 3635-3668
2. Grabowski G.A. Gaucher disease: lessons from a decade of therapy. J Pediatr 144 (2004) S15-S19
3. Grabowski G.A., Andria G., Baldellou A., Campbell P.E., Charrow J., Cohen I.J., et al. Pediatric non-neuronopathic Gaucher disease: presentation, diagnosis and assessmentConsensus statements. Eur J Pediatr 163 (2004) 58-66
4. Damiano A.M., Pastores G.M., and Ware Jr. 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 7 (1998) 373-386
5. Weinreb N.J., Charrow J., Andersson H.C., Kaplan P., Kolodny E.H., Mistry P., et al. 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. Am J Med 113 (2002) 112-119
6. Charrow J., Andersson H.C., Kaplan P., Kolodny E.H., Mistry P., Pastores G., et al. Enzyme replacement therapy and monitoring for children with type 1 Gaucher disease: consensus recommendations. J Pediatr 144 (2004) 112-120
7. Bembi B., Ciana G., Mengel E., Terk M.R., Martini C., and Wenstrup R.J. Bone complications in children with Gaucher disease. Br J Radiol 75 Suppl 1 (2002) A37-A44
8. Kaplan P., Mazur A., Manor O., Charrow J., Esplin J., Gribble T.J., et al. Acceleration of retarded growth in children with Gaucher disease after treatment with alglucerase. J Pediatr 129 (1996) 149-153
9. Elstein D., Abrahamov A., Hadas-Halpern I., and Zimran A. Withdrawal of enzyme replacement therapy in Gaucher's disease. Br J Haematol 110 (2000) 488-492
10. vom Dahl S., Poll L.W., and Haussinger D. Clinical monitoring after cessation of enzyme replacement therapy in M. Gaucher. Br J Haematol 113 (2001) 1084-1087
11. Czartoryska B., Tylki-Szymanska A., and Lugowska A. Changes in serum chitotriosidase activity with cessation of replacement enzyme (cerebrosidase) administration in Gaucher disease. Clin Biochem 33 (2000) 147-149
12. Schwartz I.V.D., Karam S., Ashton-Prolla P., Michelin K., Coelho J., Pires R.F., et al. Effects of imilglucerase withdrawal on an adult with Gaucher disease. Br J Haematol 113 (2001) 1089
13. Weinreb N.J. Interruption in enzyme replacement therapy for Gaucher disease. Br J Haematol 113 (2001) 1087-1088
14. Grinzaid K.A., Geller E., Hanna S.L., and Elsas II L.J. Cessation of enzyme replacement therapy in Gaucher disease. Genet Med 4 (2002) 427-433
15. Toth J., Erdos M., and Marodi L. Rebound hepatosplenomegaly in type 1 Gaucher disease. Eur J Haematol 70 (2003) 125-128
16. Pastores G.M., Weinreb N.J., Aerts H., Andria G., Cox T.M., Giralt M., et al. Therapeutic goals in the treatment of Gaucher disease. Semin Hematol 41 (2004) 4-14
17. Baldellou A., Andria G., Campbell P.E., Charrow J., Cohen I.J., Grabowski G.A., et al. Paediatric non-neuronopathic Gaucher disease: recommendations for treatment and monitoring. Eur J Pediatr 163 (2004) 67-75