Cyclosporine A in Ullrich Congenital Muscular Dystrophy: Long-Term Results

Table 3

Body composition evaluation: BMI and DXA. Body fat was estimated with the body mass index (BMI) and DXA. The BMI number using the CDC BMI-for-age growth charts for girls and boys is translated into a percentile that categorizes the weight status. Two patients (P3 and P6) remained in the underweight category during the study, one moved from the overweight to the obese (P 5), and 2 patients (P1 and P4) remained in the obese category at baseline and followup. However, all the patients had already a high % of body fat at baseline, including the 2 underweight. In addition, the lean mass decreased by 8.5%, while the fat mass increased by 37.6% during the treatment in 5 patients. The patients had a marked reduction of total body BMD irrespective of the level of motor function: walkers and nonwalkers had the same 𝑍 score mean value (−3.8). The BMD was also not influenced by the treatment with CsA.

Patient			DXA
	BMI (kg/m^2)		Lean Tissue Mass (kg)		Fat Tissue Mass (kg)		FAT%		BMD 𝑍 score
	Time 0	Followup	Time 0	Followup	Time 0	Followup	Time 0	Followup	Time 0	Followup
1	29.8	27.3	12.2	ND	40.7	ND	74	ND	−4.3	ND
2	15.5	23.2	9.8	10.8	18.9	40.2	63	77	−3.1	−3.9
3	13.9	12.1	8.7	ND	10.1	ND	52	ND	−4.1	ND
4	23.8	27.6	12.9	12.5	31.8	55.5	68	80	−3.6	−3.7
5	20.4	22.4	8.6	7.1	22.8	30.7	70	79	−4.6	−3.3
6	11.1	13.2	8.8	6.6	4.1	8.1	31	53	−3.1	−3.6
Mean	19.1	21.2	10.0*	9.2*	19.4*	33.6*	58*	72.2*	−3.6*	−3.6*

BMI: body mass index; DXA: dual-energy X-ray absorptiometry; BMD: bone mineral density (g/cm²); ND: not done; *: mean of DXA data from patients 2, 4, 5, 6.