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

Table 2

Study results. The three patients enrolled in the original pilot trial (P1, P2, and P3) received CsA for 2 to 3.2 years and the three new patients for 1 to 1.2 years. Megalimbs increased in all but P5. The distance walked in 6 minutes remained the same in the 2 patients able to walk. Forced vital capacity declined in all from 57.1% predicted to 45.2% predicted.

Patient	Age at treatment (Years)	Age at Followup (Years)	Megalimbs (Newton)		6 MWD (meter)		FVC %
			Time 0	Followup	Time 0	Followup	Time 0	Followup
1	9.5	12.7	218	283	NA	NA	47	24
2	9.8	11.6	106	142	NA	NA	44	32
3	8.9	10.9	106	120	NA	NA	NA	NA
4	9.0	10.2	140	169	NA	NA	41.7	31
5	9.1	10.1	241	222	189	188	59	51
6	5.5	6.7	172	236	300	304	94	88
Mean	8.6	10.4	163.8	195.3	244.5	246	57.1	45.2

Megalimbs: sum of bilateral muscle strength of elbow flexion, hand grip, knee flexion, and knee extension; 6 MWD: distance walked in 6 minutes; FVC %, forced vital capacity % of predicted; NA: not able.