Patient-reported outcomes measures of X-linked hypophosphataemia participants: findings from a prospective cohort study in the UK

Cole, Sophie; Sanchez-Santos, Maria T.; Kolovos, Spyros; Javaid, Muhammad Kassim; Pinedo-Villanueva, Rafael

doi:10.1186/s13023-023-02620-w

Orphanet Journal of Rare Diseases

Table 1 Cross-sectional summary statistics, by sex

From: Patient-reported outcomes measures of X-linked hypophosphataemia participants: findings from a prospective cohort study in the UK

Overall instrument score	Total					Female				Male
Overall instrument score	n	Mean	SD	Median	Range of submission dates	n	Mean	SD	Median	n	Mean	SD	Median	T-test P-value
Health related quality of life
EQ-5D-5L Index score	48	0.651	0.270	0.734	07/2014–08/2019	37	0.645	0.261	0.730	11	0.669	0.312	0.758	0.826
EQ5D VAS score	48	62.48	25.74	70.00	07/2014–08/2019	37	61.32	25.74	70.00	11	66.36	26.57	70.00	0.586
EQ5D-3L Index score (crosswalk)	48	0.554	0.300	0.654	07/2014–08/2019	37	0.550	0.285	0.654	11	0.567	0.361	0.642	0.887
SF-36- physical component score	44	32.67	14.19	31.66	07/2014–08/2019	35	31.31	11.96	31.19	9	37.98	20.82	36.27	0.379
SF-36- mental component score	44	48.38	10.41	49.28	07/2014–08/2019	35	48.50	11.19	50.28	9	47.89	7.14	47.82	0.843
Fatigue
FSS score	23	32.83	14.04	30.00	09/2018–08/2019	20	33.40	13.22	31.50	3	29.00	21.93	20.00	0.764
FACIT-F score	43	104.40	29.31	108.00	04/2015–08/2019	35	102.70	30.01	106.00	8	111.90	26.47	115.00	0.404
FACIT-F- physical well-being	43	18.52	6.30	20.00	04/2015–08/2019	35	18.49	6.31	20.00	8	18.75	7.11	20.50	0.921
FACIT-F- social well-being	43	20.59	5.15	22.00	04/2015–08/2019	35	20.30	5.47	21.00	8	21.65	3.97	22.58	0.451
FACIT-F- emotional well-being	43	15.86	4.67	16.00	04/2015–08/2019	35	15.63	4.74	15.00	8	16.50	4.78	17.00	0.685
FACIT-F- functional well-being	43	18.25	5.49	18.00	04/2015–08/2019	35	17.97	5.58	18.00	8	19.12	5.64	19.50	0.637
FACIT-F- fatigue subscale	43	31.33	13.28	33.00	04/2015–08/2019	35	30.29	13.37	33.00	8	35.88	12.69	38.00	0.290
Pain
SF-MPQ-2 score	46	1.94	1.51	1.77	07/2014–08/2019	37	2.05	1.52	1.86	9	1.46	1.47	0.73	0.304
painDETECT score	43	9.28	6.25	9.00	07–2014-08/2019	36	9.39	6.29	9.00	7	8.71	6.55	9.00	0.808
Negative, n (%)	28	65.12				23	63.89			5	71.43
Unclear, n (%)	12	27.91				11	30.56			1	14.29
Positive, n (%)	3	6.98				2	5.56			1	14.29
Sleep
ESS score	23	5.87	3.68	5.00	09/2018–08/2019	20	6.25	3.57	5.50	3	3.33	4.16	2.00	0.349
PSQI score	41	8.88	4.12	9.00	07/2014–06/2019	32	9.56	4.05	9.50	9	6.44	3.61	5.00	0.043
Mental wellbeing
HADS Anxiety score	41	6.17	3.83	6.00	04/2015–06/2019	32	6.28	3.56	6.00	9	5.78	4.89	7.00	0.779
Normal, n (%)	26	63.41				21	65.62			5	55.56
Mild, n (%)	10	24.39				8	25.00			2	22.22
Moderate/severe, n (%)	5	12.20				3	9.38			2	22.22
HADS Depression score	41	4.71	3.49	4.00	04/2015–06/2019	32	4.69	3.32	4.00	9	4.78	4.27	4.00	0.954
Normal, n (%)	35	85.37				28	87.50			7	77.78
Mild, n (%)	2	4.88				1	3.12			1	11.11
Moderate/severe, n (%)	4	9.76				3	9.38			1	11.11

The sample is the first submitted instrument from each patient
EQ-5D-5L (3L), EuroQol 5 dimensions-5 levels (3 levels); VAS, Visual analogue scale; SF-36, Short-form 36; PCS, Physical Component Score; MCS, Mental Component Score; FSS, Fatigue Severity Scale; ESS, Epworth Sleepiness scale; PSQI, Pittsburgh sleep quality index; FACIT-F, Functional assessment of chronic illness therapy-fatigue; SF-MPQ-2, Short form McGill pain questionnaire version 2; HADS, Hospital anxiety and depression scale

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ISSN: 1750-1172

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