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Table 4 Mortality of 99 Dutch Pompe patients compared to general Dutch population

From: Survival and associated factors in 268 adults with Pompe disease prior to treatment with enzyme replacement therapy

Analysis

Median follow-up time (range)

Observed no. deaths (O)

Expected no. deaths (E) *

Ratio (O/E)

P -value

1

2.3 (<1 month-7 years)

5

2.3

2.2

0.09

2

3.3 (<2 months-7 years)

9

2.8

3.2

0.002

  1. Analysis 1: 5 deceased patients before start of ERT Analysis 2: including 4 patients who died after start of ERT with follow-up of every patient on ERT extended with 1 year after start of ERT. * According to death probabilities derived from Dutch Central Bureau of Statistics.
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Orphanet Journal of Rare Diseases

ISSN: 1750-1172