High rate of postoperative mortality in patients with mucopolysaccharidosis I: Findings from the MPS i Registry

Journal of Pediatric Surgery

Volumn 47, Issue 3, 2012, Pages 477-484

  Arn, Pamela ^a   Whitley, Chester ^b   Wraith, J Edmond ^c   Webb, H Warner ^a   Underhill, Lisa ^d   Rangachari, Lakshmi ^d   Cox, Gerald F ^d,e  

^a NEMOURS CHILDREN S CLINIC   (United States)

^b UNIVERSITY OF MINNESOTA   (United States)

^c ST MARY S HOSPITAL   (United Kingdom)

^d GENZYME CORPORATION   (United States)

^e BOSTON CHILDREN S HOSPITAL   (United States)

Author keywords

Anesthetic complications; Hurler; Hurler Scheie; Mortality; Mucopolysaccharidosis I; Scheie; Surgery

Indexed keywords

ADOLESCENT; ADULT; ARTICLE; CENTRAL LINE PLACEMENT; CHILD; DEATH; DISEASE REGISTRY; FEMALE; HERNIOPLASTY; HUMAN; HURLER SYNDROME; INFANT; MAJOR CLINICAL STUDY; MALE; PERITONEUM VEIN SHUNT; PRIORITY JOURNAL; SPINE SURGERY; SURGICAL MORTALITY; SURGICAL TECHNIQUE; TRACHEOSTOMY; VENTRICULO PERITONEAL SHUNT;

ADOLESCENT; ADULT; CAUSE OF DEATH; CHILD; CHILD, PRESCHOOL; FEMALE; HUMANS; INFANT; INTRAOPERATIVE COMPLICATIONS; MALE; MUCOPOLYSACCHARIDOSIS I; POSTOPERATIVE COMPLICATIONS; REGISTRIES; YOUNG ADULT;

EID: 84858221318     PISSN: 00223468     EISSN: 15315037     Source Type: Journal    
DOI: 10.1016/j.jpedsurg.2011.09.042     Document Type: Article

References (36)

1. E.F. Neufeld, and J. Muenzer The mucopolysaccharidoses C. Scriver, A. Beaudet, W. Sly, The metabolic and molecular bases of inherited disease 2001 McGraw Hill New York 3421 3452
2. J. Muenzer, J.E. Wraith, and L.A. Clarke Mucopolysaccharidosis I: management and treatment guidelines Pediatrics 123 2009 19 29
3. J.J. Boelens, V. Rocha, and M. Aldenhoven Risk factor analysis of outcomes after unrelated cord blood transplantation in patients with Hurler syndrome Biol Blood Marrow Transplant 15 2009 618 625
4. J.J. Boelens, R.F. Wynn, and A. O'Meara Outcomes of hematopoietic stem cell transplantation for Hurler's syndrome in Europe: a risk factor analysis for graft failure Bone Marrow Transplant 40 2007 225 233 (Pubitemid 47108465)
5. V.K. Prasad, and J. Kurtzberg Transplant outcomes in mucopolysaccharidoses Semin Hematol 47 2010 59 69
6. R. Wynn, J. Mercer, and J. Page Use of enzyme replacement therapy (laronidase) before hematopoietic stem cell transplantation for mucopolysaccharidosis I: experience in 18 patients J Pediatr 154 2009 135 139
7. V. Valayannopoulos, J. de Blic, and N. Mahlaoui Laronidase for cardiopulmonary disease in Hurler syndrome 12 years after bone marrow transplantation Pediatrics 126 2010 e1242 e1247
8. P. Arn, J.E. Wraith, and L. Underhill Characterization of surgical procedures in patients with mucopolysaccharidosis type I: findings from the MPS I Registry J Pediatr 154 2009 859e3 864e3
9. F. Baehner, C. Schmiedeskamp, and F. Krummenauer Cumulative incidence rates of the mucopolysaccharidoses in Germany J Inherit Metab Dis 28 2005 1011 1017 (Pubitemid 43168938)
10. H.Y. Lin, S.P. Lin, and C.K. Chuang Incidence of the mucopolysaccharidoses in Taiwan, 1984-2004 Am J Med Genet A 149A 2009 960 964
11. D. Moore, M.J. Connock, and E. Wraith The prevalence of and survival in mucopolysaccharidosis I: Hurler, Hurler-Scheie and Scheie syndromes in the UK Orphanet J Rare Dis 3 2008 24
12. H. Ben Turkia, N. Tebib, and H. Azzouz Incidence of mucopolysaccharidoses in Tunisia Tunis Med 87 2009 782 785
13. M. Muñoz-Rojas, L. Bay, and L. Sanchez Clinical manifestations and treatment of mucopolysaccharidosis type I patients in Latin America as compared with the rest of the world J Inher Metab Dis 34 2011 1029 1037
14. A.M. Martins, A.P. Dualibi, and D. Norato Guidelines for the management of mucopolysaccharidosis type I J Pediatr 155 2009 S32 S46
15. G. Pastores, P. Arn, and M. Beck The MPS I registry: design, methodology, and early findings of a global disease registry for monitoring patients with mucopolysaccharidosis type I Mol Genet Metab 91 2007 37 47 (Pubitemid 46550926)
16. C. Peters, M. Balthazor, and E.G. Shapiro Outcome of unrelated donor bone marrow transplantation in 40 children with Hurler syndrome Blood 87 1996 4894 4902 (Pubitemid 26162394)
17. V.K. Prasad, A. Mendizabal, and S.H. Parikh Unrelated donor umbilical cord blood transplantation for inherited metabolic disorders in 159 pediatric patients from a single center: influence of cellular composition of the graft on transplantation outcomes Blood 112 2008 2979 2989
18. P.J. Orchard, C. Milla, and E. Braunlin Pre-transplant risk factors affecting outcome in Hurler syndrome Bone Marrow Transplant 2009
19. S. Kharbanda, A. Panoskaltsis-Mortari, and I.Y. Haddad Inflammatory cytokines and the development of pulmonary complications after allogeneic hematopoietic cell transplantation in patients with inherited metabolic storage disorders Biol Blood Marrow Transplant 12 2006 430 437 (Pubitemid 43380960)
20. A. Gassas, L. Sung, and J.J. Doyle Life-threatening pulmonary hemorrhages post bone marrow transplantation in Hurler syndrome. Report of three cases and review of the literature Bone Marrow Transplant 32 2003 213 215 (Pubitemid 36903446)
21. S.H. Ein, I. Njere, and A. Ein Six thousand three hundred sixty-one pediatric inguinal hernias: a 35-year review J Pediatr Surg 41 2006 980 986
22. J. Ghoroubi, F. Imanzadeh, and S. Askarpour Ten years study of inguinal hernia in children J Surg Pak (International) 13 2008 173 174
23. J.L. Dulucq, P. Wintringer, and A. Mahajna Laparoscopic totally extraperitoneal inguinal hernia repair: lessons learned from 3,100 hernia repairs over 15 years Surg Endosc 23 2009 482 486
24. L.A. Mermel, B.M. Farr, and R.J. Sherertz Guidelines for the management of intravascular catheter-related infections Clin Infect Dis 32 2001 1249 1272 (Pubitemid 32405897)
25. M. Pinon, S. Bezzio, and P.A. Tovo A prospective 7-year survey on central venous catheter-related complications at a single pediatric hospital Eur J Pediatr 168 2009 1505 1512
26. A.H. Yeung, M.J. Cowan, and B. Horn Airway management in children with mucopolysaccharidoses Arch Otolaryngol Head Neck Surg 135 2009 73 79
27. R. Walker, M. Darowski, and P. Morris Anaesthesia and mucopolysaccharidoses. A review of airway problems in children Anaesthesia 49 1994 1078 1084
28. I.A. Herrick, and E.J. Rhine The mucopolysaccharidoses and anaesthesia: a report of clinical experience Can J Anaesth 35 1988 67 73 (Pubitemid 18048302)
29. D. Baines, and J. Keneally Anaesthetic implications of the mucopolysaccharidoses: a fifteen-year experience in a children's hospital Anaesth Intensive Care 11 1983 198 202 (Pubitemid 13076654)
30. R.W. Walker, V. Colovic, and D.N. Robinson Postobstructive pulmonary oedema during anaesthesia in children with mucopolysaccharidoses Paediatr Anaesth 13 2003 441 447 (Pubitemid 36774027)
31. K.G. Belani, W. Krivit, and B.L. Carpenter Children with mucopolysaccharidosis: perioperative care, morbidity, mortality, and new findings J Pediatr Surg 28 1993 403 408 [discussion 8-10]
32. S.Y. Shinhar, H. Zablocki, and D.N. Madgy Airway management in mucopolysaccharide storage disorders Arch Otolaryngol Head Neck Surg 130 2004 233 237 (Pubitemid 38182554)
33. A.M. Brambrink, and U. Braun Airway management in infants and children Best Pract Res Clin Anaesthesiol 19 2005 675 697 (Pubitemid 43019590)
34. M. Aldenhoven, J.J. Boelens, and T.J. de Koning The clinical outcome of Hurler syndrome after stem cell transplantation Biol Blood Marrow Transplant 14 2008 485 498 (Pubitemid 351494741)
35. L. Clarke, J.E. Wraith, and M. Beck Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis I Pediatrics 123 2009 229 240
36. N.J. Mendelsohn, P. Harmatz, and O. Bodamer Importance of surgical history in diagnosing mucopolysaccharidosis type II (Hunter syndrome): data from the Hunter Outcome Survey Genet Med 12 2010 816 822