Table 1.

Summary of FA cohort reports

CohortLITIFARNASP*
Reporting period or date 1927-2001 1982-2001 2000 —  
Total number of subjects 1301 754 145 —  
Male-to-female ratio 1.23 1.05 1.10 ns  
Age FA diagnosed, median (range) 7 (0-48) na 5 (0-45) <.0002  
Deceased %, at time of report 38% 38% 30% ns  
Projected median survival age, years 20 24 30 —  
Leukemia, no MDS; number (% total cohort) 116 (9%) 47 (6%) 9 (6%) <.05  
Leukemia, cumulative incidence na 45% by age 50
(includes MDS) 
10% by age 24
(no MDS) 
—  
MDS, number (% total cohort) 89 (7%) 53 (7%) 23 (16%) .001  
Solid tumor, number (% total cohort) 68 (5%) 67 (9%) 13 (9%) .003  
Solid tumor, cumulative incidence na 36% by age 50 29% by age 48 — 
Liver tumor, number (% total cohort) 37 (3%) 18 (2%) 2 (1%) ns  
Hematopoietic stem cell transplant (% total cohort) 220 (17%) 219 (29%) 44 (30%) <.0001 
CohortLITIFARNASP*
Reporting period or date 1927-2001 1982-2001 2000 —  
Total number of subjects 1301 754 145 —  
Male-to-female ratio 1.23 1.05 1.10 ns  
Age FA diagnosed, median (range) 7 (0-48) na 5 (0-45) <.0002  
Deceased %, at time of report 38% 38% 30% ns  
Projected median survival age, years 20 24 30 —  
Leukemia, no MDS; number (% total cohort) 116 (9%) 47 (6%) 9 (6%) <.05  
Leukemia, cumulative incidence na 45% by age 50
(includes MDS) 
10% by age 24
(no MDS) 
—  
MDS, number (% total cohort) 89 (7%) 53 (7%) 23 (16%) .001  
Solid tumor, number (% total cohort) 68 (5%) 67 (9%) 13 (9%) .003  
Solid tumor, cumulative incidence na 36% by age 50 29% by age 48 — 
Liver tumor, number (% total cohort) 37 (3%) 18 (2%) 2 (1%) ns  
Hematopoietic stem cell transplant (% total cohort) 220 (17%) 219 (29%) 44 (30%) <.0001 

ns indicates not statistically significant; na, not available; and —, data in rows could not be subjected to test for significance.

*

P value indicates significance of data at the extremes.

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