Table 1

Late effects of BMT in SCD

Bernaudin et alVermylen et al, Brachet et alEggleston et al, Walters et alSocie et al, Ade et al10 
Pulmonary function Not reported Bronchiolitis obliterans in 2 Stable compared to baseline at least up to 3 years after BMT Restrictive defects are frequent at 3-6 months; obstructive defects are seen in 20% of patients, usually associated with cGVHD 
Growth curve “No change in mean height” Normal or improved except in 2 with cGVHD Normal if BMT was done before or after puberty 1 standard deviation score below mean height and weight in TBI based BMT; near normal mean height and weight in chemo-based BMT 
Gonadal function Male: age appropriate testosterone, LH and FSH levels. Female: primary amenorrhea in many; secondary amenorrhea in 7 Male: normal sexual maturation; small testes in all, oligospermia in 1, azospermia in another. Female: ovarian failure in 7, recovery in 2 others Male: not reported. Female: ovarian failure in 4, recovery in 2 others Male: significant reduction in spermatogenesis despite normal testosterone level. Onset of puberty usually not affected. Younger age at transplantation may have lower testicular volume and function. Female: hypergonadotrophic hypogonadism is common. Younger age at transplantation has better chance of ovarian recovery. 
Late malignancy Not reported Not reported Not reported PTLD (1% at 10 years), solid tumors (2% at 10 years); lymphoma (rare), leukemia (rare) 
Neurocognitive testing Not reported Not reported Not reported 20% of patients reported problems with memory in the first year after BMT; 10% have cognitive impairment; possible lower IQ levels 
Bernaudin et alVermylen et al, Brachet et alEggleston et al, Walters et alSocie et al, Ade et al10 
Pulmonary function Not reported Bronchiolitis obliterans in 2 Stable compared to baseline at least up to 3 years after BMT Restrictive defects are frequent at 3-6 months; obstructive defects are seen in 20% of patients, usually associated with cGVHD 
Growth curve “No change in mean height” Normal or improved except in 2 with cGVHD Normal if BMT was done before or after puberty 1 standard deviation score below mean height and weight in TBI based BMT; near normal mean height and weight in chemo-based BMT 
Gonadal function Male: age appropriate testosterone, LH and FSH levels. Female: primary amenorrhea in many; secondary amenorrhea in 7 Male: normal sexual maturation; small testes in all, oligospermia in 1, azospermia in another. Female: ovarian failure in 7, recovery in 2 others Male: not reported. Female: ovarian failure in 4, recovery in 2 others Male: significant reduction in spermatogenesis despite normal testosterone level. Onset of puberty usually not affected. Younger age at transplantation may have lower testicular volume and function. Female: hypergonadotrophic hypogonadism is common. Younger age at transplantation has better chance of ovarian recovery. 
Late malignancy Not reported Not reported Not reported PTLD (1% at 10 years), solid tumors (2% at 10 years); lymphoma (rare), leukemia (rare) 
Neurocognitive testing Not reported Not reported Not reported 20% of patients reported problems with memory in the first year after BMT; 10% have cognitive impairment; possible lower IQ levels 

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