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We compared the impact of a conditioning regimen with BU (n=16) or fractionated TBI (n=42) on height growth during adolescence and final height (FH), in 58 adults transplanted for acute leukaemia before adolescence (younger than 9 for girls and 11 for boys, and prepubertal). Heights were measured at three key periods, that is, transplantation, before adolescence, and FH, and compared using height standard deviation score (SDS) and cumulative change in SDS. The influence of the conditioning regimen was assessed using multiple linear regression and adjusting for gender, central nervous system irradiation, age and leukaemia status at transplant and type of transplantation. Overall mean height SDS was near normal at transplantation and before adolescence (0.2+/-0.1 and -0.2+/-0.1, respectively), but decreased to -1.6+/-0.1 at FH. There were significant differences between the TBI and BU groups when comparing FH SDS (-1.8+/-0.2 vs -0.8+/-0.2, P=0.001), mean change in height SDS from transplantation to FH (-2+/-0.1 vs -1.1+/-0.2, P=0.002) and mean change in height SDS during adolescence (-1.6+/-0.1 vs -0.7+/-0.2, P=0.003). We conclude that preparations involving BU, although less toxic than TBI-containing regimens, also have adverse effects on growth, predominantly during adolescence.

IntroductionTwo meta-analyses showed lower bone mineral density (BMD) in patients with haemophilia (haemophilia type and severity were often not specified) compared with healthy controls. This finding could be related to reduced mobility and sedentary lifestyle, and/or hepatitis C or HIV infection. The aim of this study is to determine osteoporosis prevalence in patients with haemophilia classified in function of the disease type (A or B) and severity, and to evaluate the potential role of regular prophylactic factor replacement (early vs delayed initiation) in preserving or restoring BMD.Methods and analysisThe haemoPHILia and ostEoporOSis Study is a prospective, controlled, multicentre study that will include patients in France (13 haemophilia treatment centres), Belgium (1 centre) and Romania (1 centre). In total, 240 patients with haemophilia and 240 matched healthy controls will be recruited (1:1). The primary objective is to determine osteoporosis prevalence in patients with severe haemophilia A and B (HA and HB) without prophylaxis, compared with healthy controls. Secondary outcomes include: prevalence of osteoporosis and osteopenia in patients with mild, moderate and severe HA or HB with prophylaxis (grouped in function of their age at prophylaxis initiation), compared with healthy subjects; BMD in patients with HA and HB of comparable severity; correlation between BMD and basal factor VIII/IX levels and thrombin potential; and quantification of plasmatic markers of bone remodelling (formation and resorption) in patients with haemophilia.Ethics and disseminationThe protocol was approved by the French Ethics Committee and by the French National Agency for Medicines and Health Products Safety (number: 2019-A03358-49). The results of this study will be actively disseminated through scientific publications and conference presentations.Trial registration numberNCT04384341.