Abstract
AbstractHematopoietic stem cell transplant (SCT) is currently the only therapy that can restore normal hematopoiesis in patients with Fanconi anemia (FA). Patients with FA have a high baseline risk of squamous cell cancers (SCCs) of the head, neck, and esophagus, and SCT conditioning may increase SCC incidence. We evaluated the risks of SCC and death in 145 patients with FA in the North American Survey (NAS) cohort who did not receive transplants, and 117 patients with FA in the Hôpital Saint Louis (SLH) cohort who did receive transplants. The age-specific hazard of SCC was 4.4-fold higher in patients who received transplants than in those who did not (P = .003), and SCCs occurred at significantly younger ages in the former (respective medians, 18 and 33 years, P = .004). Survival after SCC was similarly poor in both cohorts (P = .135, median, 13 months). The hazard of SCC increased at a greater than linear rate, to 4.4% per year by age 40 in NAS and 4.7% per year by 10 years after transplant in SLH. In SLH, the hazard of non-SCC death was biphasic, declining significantly (P = .004) from 7.1% per month during the first 6 months after transplant to 0.13% per month (1.6% per year) after the first year. Acute and chronic graft-versus-host diseases were significant SCC risk factors. Adverse event rates in these cohorts provide historical control rates to assess emerging therapies for FA.
Highlights
Introduction plantsThe age-specific hazard of squamous cell cancers (SCCs) was 4.4-fold higher in patients who received transplants than in those who did not (P ؍.003), and SCCs occurred at significantly younger ages in the former
These hypotheses were declared in our study protocol prior to analyzing the data; we describe the specific factors in “Results.” For each outcome, we considered these sets of risk factors as 3 separate families of hypotheses
In Saint Louis Transplant Cohort (SLH), 117 patients with Fanconi anemia (FA) followed after transplantation contributed 508 person-years, 48 non-SCC deaths, and 11 SCCs (Table 1)
Summary
The age-specific hazard of SCC was 4.4-fold higher in patients who received transplants than in those who did not (P ؍.003), and SCCs occurred at significantly younger ages in the former (respective medians, 18 and 33 years, P ؍.004). Survival after SCC was poor in both cohorts (P ؍.135, median, 13 months). The hazard of SCC increased at a greater than linear rate, to 4.4% per year by age 40 in NAS and 4.7% per year by 10 years after transplant in SLH. In SLH, the hazard of non-SCC death was biphasic, declining significantly (P ؍.004) from 7.1% per month during the first 6 months after transplant to 0.13% per month (1.6% per year) after the first year. Acute and chronic graft-versushost diseases were significant SCC risk factors. Adverse event rates in these cohorts provide historical control rates to assess emerging therapies for FA. (Blood. 2005;105:67-73)
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