Laronidase for cardiopulmonary disease in Hurler syndrome 12 years after bone marrow transplantation

Pediatrics

Volumn 126, Issue 5, 2010, Pages

  Valayannopoulos, Vassili ^a   De Blic, Jacques ^a   Mahlaoui, Nizar ^a   Stos, Bertrand ^a   Jaubert, Francis ^a   Bonnet, Damien ^a   Fischer, Alain ^a   De Lonlay, Pascale ^a  

^a HÔPITAL NECKER ENFANTS MALADES   (France)

Author keywords

Enzyme replacement therapy; Hematopoietic stem cell transplantation; Hurler syndrome; Interstitial lung disease; Laronidase; Mucopolysaccharidosis type I; Pulmonary arterial hypertension

Indexed keywords

BUSULFAN; CARBON MONOXIDE; CYCLOPHOSPHAMIDE; GLYCOSAMINOGLYCAN; LARONIDASE; LEVO IDURONIDASE; OXYGEN;

ADOLESCENT; ARTERIAL GAS; ARTICLE; BONE MARROW TRANSPLANTATION; BRONCHOSCOPY; CARDIOPULMONARY INSUFFICIENCY; CASE REPORT; CHILD; CLINICAL FEATURE; COMPUTER ASSISTED TOMOGRAPHY; DISEASE COURSE; DISEASE SEVERITY; ENZYME ACTIVITY; ENZYME REPLACEMENT; GENE MUTATION; GENOTYPE; GRAFT FAILURE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HETEROZYGOSITY; HISTOCOMPATIBILITY; HUMAN; HURLER SYNDROME; HYPOXEMIA; IMMUNOSUPPRESSIVE TREATMENT; INTERSTITIAL LUNG DISEASE; LUNG ALVEOLUS WALL; LUNG ARTERY PRESSURE; LUNG BIOPSY; LUNG FUNCTION TEST; LUNG INFILTRATE; LUNG VENTILATION; OXYGEN THERAPY; POLYSOMNOGRAPHY; POSITIVE END EXPIRATORY PRESSURE; PRESCHOOL CHILD; PRIORITY JOURNAL; PULMONARY HYPERTENSION; QUALITY OF LIFE; RESPIRATORY ACIDOSIS; RESPIRATORY FAILURE; SCHOOL CHILD; SLEEP APNEA SYNDROME; TACHYPNEA; UPPER RESPIRATORY TRACT OBSTRUCTION; VITAL CAPACITY; WHEELCHAIR;

ADOLESCENT; BONE MARROW TRANSPLANTATION; CHILD; CHILD, PRESCHOOL; COMBINED MODALITY THERAPY; CONTINUOUS POSITIVE AIRWAY PRESSURE; FOLLOW-UP STUDIES; GENOTYPE; HETEROZYGOTE DETECTION; HISTOCOMPATIBILITY TESTING; HUMANS; HYPERTENSION, PULMONARY; IDURONIDASE; INFANT; INFUSIONS, INTRAVENOUS; LUNG DISEASES, INTERSTITIAL; MALE; MUCOPOLYSACCHARIDOSIS I; OXYGEN INHALATION THERAPY; PHENOTYPE; PULMONARY ALVEOLI; QUALITY OF LIFE; RECOMBINANT PROTEINS; RESPIRATORY INSUFFICIENCY; SLEEP APNEA, OBSTRUCTIVE;

EID: 78049428687     PISSN: 00314005     EISSN: 10984275     Source Type: Journal    
DOI: 10.1542/peds.2009-2843     Document Type: Article

References (27)

1. Peters C, Shapiro EG, Anderson J, et al. Hurler syndrome: II. Outcome of HLA-genotypically identical sibling and HLA-haploidentical related donor bone marrow transplantation in fifty-four children. The Storage Disease Collaborative Study Group. Blood. 1998;91(7):2601-2608
2. Peters C, Shapiro EG, Krivit W. Hurler syndrome: past, present, and future. J Pediatr. 1998;133(1):7-9
3. Staba SL, Escolar ML, Poe M, et al. Cordblood transplants from unrelated donors in patients with Hurler's syndrome. N Engl J Med. 2004;350(19):1960-1969
4. Boelens JJ, Wynn RF, O'Meara A, et al. Outcomes of hematopoietic stem cell transplantation for Hurler's syndrome in Europe: a risk factor analysis for graft failure. Bone Marrow Transplant. 2007;40(3):225-233
5. Kakkis ED, Muenzer J, Tiller GE, et al. Enzyme-replacement therapy in mucopolysaccharidosis I. N Engl J Med. 2001;344(3):182-188
6. Wraith JE, Clarke LA, Beck M, et al. Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase). J Pediatr. 2004;144(5):581-588
7. Sifuentes M, Doroshow R, Hoft R, et al. A follow-up study of MPS I patients treated with laronidase enzyme replacement therapy for 6 years. Mol Genet Metab. 2007;90(2):171-180 (Pubitemid 46108607)
8. Wraith JE, Beck M, Lane R, et al. Enzyme replacement therapy in patients who have mucopolysaccharidosis I and are younger than 5 years: results of a multinational study of recombinant human α-L-iduronidase (laronidase). Pediatrics. 2007;120(1). Available at: www.pediatrics.org/cgi/content/full/120/ 1/e37
9. Clarke L, Wraith J, Beck M, et al. Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis I. Pediatrics. 2009;123(1):229-240
10. Grewal SS, Wynn R, Abdenur JE, et al. Safety and efficacy of enzyme replacement therapy in combination with hematopoietic stem cell transplantation in Hurler syndrome. Genet Med. 2005;7(2):143-146
11. Cox-Brinkman J, Boelens JJ, Wraith JE, et al. Haematopoietic cell transplantation (HCT) in combination with enzyme replacement therapy (ERT) in patients with Hurler syndrome. Bone Marrow Transplant. 2006;38(1):17-21
12. Hirth A, Berg A, Greve G. Successful treatment of severe heart failure in an infant with Hurler syndrome. J Inherit Metab Dis. 2007;30(5):820
13. Tolar J, Grewal SS, Bjoraker KJ, et al. Combination of enzyme replacement and hematopoietic stem cell transplantation as therapy for Hurler syndrome. Bone Marrow Transplant. 2008;41(6):531-535 (Pubitemid 351472144)
14. Guffon N, Souillet G, Maire I, Straczek J, Guibaud P. Follow-up of nine patients with Hurler syndrome after bone marrow transplantation. J Pediatr. 1998;133(1):119-125
15. Aldenhoven M, Boelens JJ, de Koning TJ. The clinical outcome of Hurler syndrome after stem cell transplantation. Biol Blood Marrow Transplant. 2008;14(5):485-498
16. Connell P, McCreery K, Doyle A, Darcy F, O'Meara A, Brosnahan D. Central corneal thickness and its relationship to intraocular pressure in mucopolysaccararidoses-1 following bone marrow transplantation. J AAPOS. 2008;12(1):7-10
17. Taylor C, Brady P, O'Meara A, Moore D, Dowling F, Fogarty E. Mobility in Hurler syndrome. J Pediatr Orthop. 2008;28(2):163-168
18. Malm G, Gustafsson B, Berglund G, et al. Outcome in six children with mucopolysaccharidosis type IH, Hurler syndrome, after haematopoietic stem cell transplantation (HSCT). Acta Paediatr. 2008;97(8):1108-1112
19. Pouchot J, Ruperto N, Lemelle I, et al. The French version of the Childhood Health Assessment Questionnaire (CHAQ) and the Child Health Questionnaire (CHQ). Clin Exp Rheumatol. 2001;19(4 suppl 23):S60-S65 (Pubitemid 33730189)
20. Kosinski M, Zhao SZ, Dedhiya S, Osterhaus JT, Ware JE Jr. Determining minimally important changes in generic and disease-specific health-related quality of life questionnaires in clinical trials of rheumatoid arthritis. Arthritis Rheum. 2000;43(7):1478-1487
21. Giugliani R, Muñoz Rojas V, Martins A, et al. A dose-optimization trial of laronidase (Aldurazyme) in patients with mucopolysaccharidosis I. Mol Genet Metab. 2009;96(1):13-19
22. Gal A. Drug and radiation toxicity. In: Tomashefski J, ed. Dail and Hammar's Pulmonary Pathology. 3rd ed. New York, NY: Springer; 2008:807-830
23. Tolar J, Orchard PJ, Key NS, Blazar BR. Circulating anticoagulant glycosaminoglycans in mucopolysaccharidosis type I. J Thromb Haemost. 2008;6(5):893-895
24. Levine DS, Navarro OM, Chaudry G, Doyle JJ, Blaser SI. Imaging the complications of bone marrow transplantation in children. Radiographics. 2007;27(2):307-324
25. Limsuwan A, Pakakasama S, Rochanawutanon M, Hong-eng S. Pulmonary arterial hypertension after childhood cancer therapy and bone marrow transplantation. Cardiology. 2006;105(3):188-194
26. Passage MB, Krieger AW, Peinovich MC, et al. Continuous infusion of enzyme replacement therapy is inferior to weekly infusions in MPS I dogs. J Inherit Metab Dis. 2009; In press
27. Khanna G, Van Heest AE, Agel J, et al. Analysis of factors affecting development of carpal tunnel syndrome in patients with Hurler syndrome after hematopoietic cell transplantation. Bone Marrow Transplant. 2007;39(6):331-334