Spinal Muscular Atrophy Type I: Is It Ethical to Standardize Supportive Care Intervention in Clinical Trials?

Journal of Child Neurology

Volumn 32, Issue 2, 2017, Pages 155-160

  Finkel, Richard S ^a   Bishop, Kathie M ^b   Nelson, Robert M ^c  

^a NEMOURS CHILDREN S HOSPITAL   (United States)

^b Tioga Pharmaceuticals Inc   (United States)

^c CENTER FOR DRUG EVALUATION AND RESEARCH   (United States)

Author keywords

clinical trial design; SMA; standard of care; Werdnig Hoffmann disease

Indexed keywords

ARTICLE; ASIA; DATA ANALYSIS; EUROPE; HUMAN; NORTH AMERICA; NUTRITIONAL SUPPORT; OUTCOME ASSESSMENT; PHYSICIAN; PHYSIOTHERAPIST; PRIORITY JOURNAL; SPINAL MUSCULAR ATROPHY; STATISTICAL ANALYSIS; CLINICAL TRIAL (TOPIC); ETHICS; HEREDITARY SPINAL MUSCULAR ATROPHY; INFANT; INTERNATIONAL COOPERATION; METHODOLOGY; STANDARDS;

CLINICAL TRIALS AS TOPIC; HUMANS; INFANT; INTERNATIONALITY; RESEARCH DESIGN; SPINAL MUSCULAR ATROPHIES OF CHILDHOOD;

EID: 85011585341     PISSN: 08830738     EISSN: 17088283     Source Type: Journal    
DOI: 10.1177/0883073816671236     Document Type: Article

References (18)

1. Zerres K, Rudnik-Schoneborn S,. Natural history in proximal spinal muscular atrophy. Clinical analysis of 445 patients and suggestions for a modification of existing classifications. Arch Neurol. 1995; 52: 518-523.
2. Oskoui M, Levy G, Garland CJ, et al. The changing natural history of spinal muscular atrophy type 1. Neurology. 2007; 69: 1931-1936.
3. Feldkotter M, Schwarzer V, Wirth R, Wienker TF, Wirth B,. Quantitative analyses of SMN1 and SMN2 based on real-time lightCycler PCR: fast and highly reliable carrier testing and prediction of severity of spinal muscular atrophy. Am J Human Genet. 2002; 70: 358-368.
4. Finkel RS, McDermott MP, Kaufmann P, et al. Observational study of spinal muscular atrophy type I and implications for clinical trials. Neurology. 2014; 83: 810-817.
5. Swoboda KJ, Prior TW, Scott CB, et al. Natural history of denervation in SMA: relation to age, SMN2 copy number, and function. Ann Neurol. 2005; 57: 704-712.
6. Wang CH, Finkel RS, Bertini ES, et al. Consensus statement for standard of care in spinal muscular atrophy. J Child Neurol. 2007; 22: 1027-1049.
7. Rigo F, Hua Y, Krainer AR, Bennett CF,. Antisense-based therapy for the treatment of spinal muscular atrophy. J Cell Biol. 2012; 199: 21-25.
8. Chopra JS,. Neurological consequences of protein and protein-calorie undernutrition. Crit Rev Neurobiol. 1991; 6: 99-117.
9. Tassie B, Isaacs D, Kilham H, Kerridge I,. Management of children with spinal muscular atrophy type 1 in Australia. J Paediatr Child Health. 2013; 49: 815-819.
10. Durkin ET, Schroth MK, Helin M, Shaaban AF,. Early laparoscopic fundoplication and gastrostomy in infants with spinal muscular atrophy type I. J Pediatr Surg. 2008; 43: 2031-2037.
11. Roper H, Quinlivan R, Workshop P,. Implementation of "the consensus statement for the standard of care in spinal muscular atrophy" when applied to infants with severe type 1 SMA in the UK. Arch Dis Child. 2010; 95: 845-849.
12. Cuisset JM, Estournet B, French Ministry of Health. Recommendations for the diagnosis and management of typical childhood spinal muscular atrophy. Rev Neurol. 2012; 168: 902-909.
13. Mitchell I,. Spinal muscular atrophy type 1: what are the ethics and practicality of respiratory support? Paediatr Respir Rev. 2006; 7 (suppl 1): S210-S211.
14. Finkel R, Bertini E, Muntoni F, Mercuri E,. 209th ENMC International Workshop: Outcome Measures and Clinical Trial Readiness in Spinal Muscular Atrophy 7-9 November 2014, Heemskerk, the Netherlands. Neuromuscul Disord. 2015; 25: 593-602.
15. Roth-Cline M, Gerson J, Bright P, Lee CS, Nelson RM,. Ethical considerations in conducting pediatric research. Handb Exp Pharmacol. 2011; 205: 219-244.
16. Lidz CW, Albert K, Appelbaum P, Dunn LB, Overton E, Pivovarova E,. Why is therapeutic misconception so prevalent? Camb Q Health Ethics. 2015; 24: 231-241.
17. Wang CH, Finkel RS, Bertini ES, et al. Consensus statement for standard of care in spinal muscular atrophy. J Child Neurol. 2007; 22: 1027-1049.
18. European Neuromuscular Centre, 218th ENMC International Workshop: "Eight years on, revisiting the Consensus Statement of Care in SMA," February 19-21, 2016. enmc.org.