Long-term efficacy and safety of sirolimus therapy in patients with lymphangioleiomyomatosis

Hu, Siqi; Wu, Xiuxiu; Xu, Wenshuai; Tian, Xinlun; Yang, Yanli; Wang, Shao-Ting; Liu, Song; Xu, Xingxiang; Xu, Kai-Feng

doi:10.1186/s13023-019-1178-2

Orphanet Journal of Rare Diseases

Table 3 Paired comparisons of functional tests and serum VEGF-D levels between the baseline and annual time points during sirolimus treatment

From: Long-term efficacy and safety of sirolimus therapy in patients with lymphangioleiomyomatosis

Variable	1 year			2 years			3 years			4 years
Variable	Baseline	After sirolimus	P value	Baseline	After sirolimus	P value	Baseline	After sirolimus	P value	Baseline	After sirolimus	P value
Pulmonary ventilation function	N = 67			N = 36			N = 22			N = 11
FEV₁ (ml)	1590 ± 690	1640 ± 710	0.045	1640 ± 610	1610 ± 690	0.462	1470 ± 660	1520 ± 740	0.560	1440 ± 590	1310 ± 440	0.099
FVC (ml)	2640 ± 760	2830 ± 700	0.004	2700 ± 620	2880 ± 560	0.044	2680 ± 660	2780 ± 630	0.398	2570 ± 560	2800 ± 380	0.149
FEV₁%pred	55.97 ± 23.75	59.28 ± 24.3	0.004	57.48 ± 21.97	58.28 ± 24.6	0.705	54.22 ± 23	57.02 ± 26.44	0.346	50.38 ± 21.09	49.19 ± 17.75	0.557
FVC%pred	80.85 ± 22.37	87.87 ± 24.06	0.005	83.2 ± 17.94	89.68 ± 18.03	0.019	83.56 ± 22.27	92.08 ± 22.08	0.054	81.32 ± 18.72	90.84 ± 15.3	0.128
FEV₁/FVC	56.27 ± 17.42	56.33 ± 18.02	0.966	58.32 ± 20.66	53.72 ± 21.82	0.000	53.59 ± 22.1	52.94 ± 23.07	0.812	54.04 ± 19.9	45.25 ± 15.17	0.014
Gas exchange	N = 48			N = 26			N = 19			N = 7
DLCO%pred	40.62 ± 20.34	41.23 ± 20.42	0.378	46.94 ± 23.87	45.23 ± 22.26	0.197	32.86 ± 19.01	35.64 ± 16.91	0.104	34.46 ± 17.65	30.49 ± 12.71	0.109
Arterial blood gas	N = 69			N = 39			N = 31			N = 17
PaO₂ (mmHg)	66.99 ± 12.58	71.21 ± 14.98	0.006	68.14 ± 12.61	75.68 ± 16.5	< 0.001	66.34 ± 10.68	70.63 ± 13.36	0.026	69.24 ± 9.662	74.09 ± 11.02	0.084
P(A-a) O₂ (mmHg)	41.29 ± 13.51	36.67 ± 16.07	0.013	40.6 ± 13.28	32.97 ± 17.23	< 0.001	42.63 ± 12.13	36.76 ± 13.89	0.007	37.89 ± 12.72	35.61 ± 11.6	0.504
6-min walk test	N = 67			N = 34			N = 27			N = 12
6MWD (m)	417 ± 122	461 ± 105	< 0.001	420 ± 120	472 ± 98	< 0.001	408 ± 105	459 ± 107	0.004	379 ± 145	436 ± 96	0.1572
SGRQ	N = 63			N = 33			N = 27			N = 12
Total score	46.11 ± 22.11	38.37 ± 22	< 0.001	42.85 ± 21.83	31.21 ± 21.79	< 0.001	48.04 ± 23.24	38.93 ± 21.94	0.0078	53.25 ± 24.51	49.08 ± 21.95	0.5019
Serum VEGF-D level	N = 89			N = 50			N = 34			N = 15
VEGF-D (pg/ml)	3594 ± 3156	2001 ± 1972	< 0.001	3711 ± 3714	2133 ± 2550	< 0.001	3280 ± 2228	1761 ± 1255	< 0.001	3445 ± 2453	1851 ± 1355	< 0.001

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ISSN: 1750-1172

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