Table 2.

Late Effects after HCT in Patients with FA

Reference	Number	Main Findings
Bonfim et al. [36]	157	Population was a median 9 years in follow-up mostly conditioned without radiation, with HCT from a matched related donor, and with chronic GVHD in about one-third. Late effects included malignancy in 15, hypothyroidism in 29, gonadal dysfunction in 49, neurologic dysfunction in 25, and hearing loss in 15.
Rosenberg et al. [15]	117	Population had 508 person-years of follow-up mostly conditioned with radiation and one-half with HCT from a matched related donor and one-half from a matched unrelated donor. Late effects included non-SCC deaths in 48 and SCC in 11. Risk of SCC was 4.4-fold higher after HCT versus FA patients without HCT and SCC occurred at younger ages. GVHD was identified as a significant risk factor.
Barnum et al. [50]	44	Population was a median 3.1 years in follow-up mostly conditioned with radiation, with HCT from a matched unrelated donor or cord blood, and with chronic GVHD in 5%. Late effects included hypothyroidism in 57%, gonadal dysfunction in 27%, short stature in 50%, vitamin D deficiency in 71%, insulin resistance in 38%, and low bone mineral density in 43%.
Rose et al. [51]	39	Population details were limited, though most were conditioned with radiation. Identified endocrine issues in those after HCT included hypothyroidism in 65% of children and 43% of adults, growth hormone deficiency in 22% of children and 25% of adults, gonadal dysfunction in 40% of children and 43% of adults, hyperglycemia in 30% of children and 9% of adults, and low bone mineral density in 13% of children and 12% of adults.
Anur et al. [39]	22	Population was a median 7.4 years in follow-up mostly conditioned with radiation, with HCT from an alternative donor, and without chronic GVHD reported. Late effects included malignancy in 2, hypothyroidism in 10, hyperglycemia in 10, gonadal dysfunction in 12, cataracts in 4, and hearing loss in 5. TBI was a risk factor for late effects.
Petryk et al. [52]	20	Population was a median 2.8 years in follow-up all conditioned with radiation and with chronic GVHD in 15%. Late effects included lower height and body mass index than controls, lower lean body mass, hypothyroidism in 6, gonadal dysfunction in 3, and lower bone mineral density than controls.
Sanders et al. [53]	15	Population was a median 20 years in follow-up mostly conditioned without radiation, with HCT from a matched related donor, and with chronic GVHD in about one-third. Late effects included malignancy in 2 with other reported outcomes mixed in with non-FA aplastic anemia patients.