Procedures for Comparing Samples with Multiple Endpoints

O’Brien, Peter C.

doi:10.2307/2531158

Cited by 1,059 publications

(742 citation statements)

References 7 publications

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“…13 FVC was measured by spirometry according to standard methods, using the best effort of three determinations as the measure of FVC for that day. 14,15 Two tests, typically one day apart, were performed during each evaluation and the best effort was used as the result for all analyses.…”

Section: Efficacy Assessmentmentioning

confidence: 99%

A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome)

Muenzer

Wraith

Beck³

et al. 2006

Genetics in Medicine

521

509

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Purpose: To evaluate the safety and efficacy of recombinant human iduronate-2-sulfatase (idursulfase) in the treatment of mucopolysaccharidosis II. Methods: Ninety-six mucopolysaccharidosis II patients between 5 and 31 years of age were enrolled in a double-blind, placebo-controlled trial. Patients were randomized to placebo infusions, weekly idursulfase (0.5 mg/kg) infusions or every-other-week infusions of idursulfase (0.5 mg/kg).Efficacy was evaluated using a composite endpoint consisting of distance walked in 6 minutes and the percentage of predicted forced vital capacity based on the sum of the ranks of change from baseline. Results: Patients in the weekly and every-other-week idursulfase groups exhibited significant improvement in the composite endpoint compared to placebo (P ϭ 0.0049 for weekly and P ϭ 0.0416 for every-other-week) after one year. The weekly dosing group experienced a 37-m increase in the 6-minute-walk distance (P ϭ 0.013), a 2.7% increase in percentage of predicted forced vital capacity (P ϭ 0.065), and a 160 mL increase in absolute forced vital capacity (P ϭ 0.001) compared to placebo group at 53 weeks. Idursulfase was generally well tolerated, but infusion reactions did occur.

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Section: Efficacy Assessmentmentioning

confidence: 99%

A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome)

Muenzer

Wraith

Beck³

et al. 2006

Genetics in Medicine

521

509

View full text Add to dashboard Cite

show abstract

“…The composite score was created by calculating individual z-scores for each test and then summing the scores, an approach we have used previously (21,22) and that is recommended over modeling each predictor individually because it increases statistical power and reduces type-I error (23,24). Because age was confounded with native disease (patients with cystic fibrosis [CF] are younger than patients without CF), we did not control for age in our primary analyses of neurocognitive function.…”

Section: Methodsmentioning

confidence: 99%

Neurocognitive Changes after Lung Transplantation

Smith¹,

Rivelli²,

Waters³

et al. 2014

Annals ATS

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“…The comparisons were also made between respective good and poor recovery subgroups of the two groups employing global test statistics. 14 …”

Section: Discussionmentioning

confidence: 99%

Is methyl prednisolone useful in acute transverse myelitis?

Kalita

2001

Spinal Cord

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Study design: Hospital based observational study. Objectives: To evaluate the role of methyl prednisolone (MPS) in the management of acute transverse myelitis (ATM). Methods: Twenty-one patients with ATM were included in a prospective hospital based study during 1992 ± 1997. All the patients underwent neurological examination, spinal MRI, somatosensory and motor evoked potentials of both upper and lower limbs and concentric needle EMG study. Twelve consecutive patients did not receive MPS therapy who were managed during 1992 ± 1994 and nine consecutive patients during 1995 ± 1997 received MPS therapy in a dose of 500 mg i.v. for 5 days. The clinical and neurophysiological studies were repeated 3 months later. The outcome was de®ned on the basis of Barthel index (BI) score at the end of 3 months into good (BI512) and poor (BI512). Results: The age of MPS group was 25.5 years (range 12 ± 42) and three were females. The age of non MPS group was 33.5 years (range 16 ± 70) and two were females. In the MPS group 33% had poor outcome compared to 67% in the non MPS group. In the MPS group mean admission BI score was 7.3 which improved to 14.6 after MPS therapy. In the non MPS group, the admission BI score was 3.2 which improved to 9.6 at 3 month follow-up. In patients with complete paraplegia, evidence of denervation on EMG and unrecordable central motor conduction time to lower limb and tibial SEP were associated with poor outcome irrespective of MPS treatment. Global test statistics did not suggest a bene®cial role of MPS therapy in the outcome of ATM. Conclusion: Our results do not suggest a bene®cial role of methyl prednisolone on the 3 month outcome of ATM. Spinal Cord (2001) 39, 471 ± 476

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Procedures for Comparing Samples with Multiple Endpoints

Cited by 1,059 publications

References 7 publications

A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome)

A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome)

Neurocognitive Changes after Lung Transplantation

Is methyl prednisolone useful in acute transverse myelitis?

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