Young XLH Patients-Reported Experience with a Supportive Care Program

Figures & data

Table 1 Baseline Demographic and Disease Characteristics of XLH Patients Monitored in the aXess Program

	All Patients			Patients Initiating Treatment			Patients Already Undergoing Treatment
	n = 59			n = 16			n = 43
Female sex	36		61%	8		50%	28	65%
Male sex	23		39%	8		50%	15	35%
Age
Pre-puberty (2.7–10 years)	36		61%	12		75%	24	56%
Post-puberty (11–18 years)	23		39%	4		25%	19	44%
Mean - year	9.8			8.5			10.4
SD – year	3.8			3.9			3.7
Height z-score (height-for-age Z)
Mean	−0.9006			−0.8929			−0.9035
Median	−0.8742			−0.8080			−0.8923
SD	1.0686			1.1650			1.0445
Min-Max	−3.4202	1.2911		−3.2607	0.9565		−3.4202	1.2911
Lower - Upper Quartile	−1.7125	−0.2398		−1.7987	0.1152		−1.6565	−0.3390
Body mass index z-score (BMI-for-age Z)
Mean	0.6775			0.9540			0.5721
Median	0.6609			0.9695			0.6150
SD	0.7694			0.6418			0.7944
Min-Max	−1.0348		2.3734	−0.1873		2.0878	−1.0348	2.3734
Lower - Upper Quartile	0.2229		1.1600	0.3777		1.5490	−0.0125	1.0652
Schooling
In School	50		85%	12		75%	38	88%
Out-of-school	9		15%	4		25%	5	12%
Time elapsed between XLH diagnosis and start of current medical treatment
Mean - year	7.9			6.8			8.3
Time elapsed between XLH diagnosis and aXess inclusion
Mean - year	8.8			6.8			9.6
Time elapsed between start of current medical treatment and aXess inclusion
Mean – month	11.0			0.2			15.0
XLH classification
De Novo	13		22%	4		25%	9	21%
Familial	36		61%	8		50%	28	65%
Not specified	10		17%	4		25%	6	14%
Use of medications for XLH
Growth Hormones	12		20%	1		6%	11	26%

Notes: For each characteristic, the percentages are shown in bold according to the subgroups: all patients, patients initiating treatment or patients already undergoing treatment.

Abbreviations: XLH, X-linked hypophosphatemia; SD, standard deviation; BMI, body mass index.

Figure 1 Flow chart. The outline of the selection and flow of XLH patients.

Abbreviation: XLH, X-linked hypophosphatemia.

Table 2 Number of aXess Nurse Calls to XLH Patients (Subgroups of Patients Initiating Treatment and Patients Already Receiving Treatment), to Pharmacy, to Medical Analysis Laboratory, to Home Care Nurses and to Physician

Hotline	All Patients n=59	Patients Initiating Treatment n=16		Patients Already Undergoing Treatment n=43
	n	n	%	n	%
Patient	625	278	44%	347	56%
Programmed follow-up nurse calls	241	104	43%	137	57%
Average number of programmed follow-up nurse calls / patient	4.1	6.5		3.2
Patient-requested calls	384	174	45%	210	55%
Coordination with pharmacy	205	61	30%	144	70%
Coordination with medical analysis laboratory	66	42	64%	24	36%
Coordination with home care nurse	34	27	79%	7	21%
Coordination with physician	29	19	66%	10	34%
Pharmacovigilance	31	12	39%	19	61%
Confinement (COVID-19)	19	13	68%	6	32%
Pharmacist	261	129	49%	132	51%
Home care nurse	212	114	54%	98	46%
Medical analysis laboratory	95	72	76%	23	24%
Prescribing physician	59	30	51%	29	49%

Notes: XLH patient calls were detailed in the table The calls from the aXess nurses are differentiated according to the type of profile of the callers (highlighted in grey and bold in the table). For patients, calls scheduled as part of the patient support program are distinguished from calls requested by the patient (in bold).

Table 3 Change from Baseline (D0) to 6 and 12 Months in the Mean PedSQL Generic Core Scales (Global and Sub-Domains Physical, Emotional, Social, School Functioning) for All Patients

	All Patients
	D0	M6	M12
	n=59	n=49	n=35
SCORE TOTAL PedsQL (Mean ± SD)	75.62 ± 0.31	82.11* ± 0.27	85.35* ± 0.24
SCORE PedsQL/Physical Functioning	79.42 ± 0.31	82.07 ± 0.28	89.37* ± 0.20
SCORE PedsQL/Emotional Functioning	65.75 ± 0.31	75.51* ± 0.28	74.85* ± 0.30
SCORE PedsQL/Social Functioning	80.58 ± 0.30	89.48* ± 0.21	89.71* ± 0.21
SCORE PedsQL/School Functioning	74.37 ± 0.31	81.33* ± 0.27	85.05* ± 0.24

Notes: Values marked with *Denotes statistically significant difference (p < 0.05 for all changes from baseline). The overall quality of life score is shown in bold and the score for each subdomain of the quality of life measure is shown.

Abbreviation: SD, standard deviation.

Table 4 Change from Baseline to 6 and 12 Months in the Mean PedSQL Generic Core Scales (Global and Sub-Domains Physical, Emotional, Social, School Functioning), Subgroup of Patients Initiating Treatment

	Patients Initiating Treatment
	D0	M6	M12
	n=16	n=10	n=6
SCORE TOTAL PedsQL (Mean ± SD)	71.28 ± 0.32	79.79* ± 0.31	76.63* ± 0.30
SCORE PedsQL/Physical Functioning	77.53 ± 0.29	88.43* ± 0.24	87.50 ± 0.21
SCORE PedsQL/Emotional Functioning	55.31 ± 0.35	68.50* ± 0.35	58.33 ± 0.37
SCORE PedsQL/Social Functioning	75.93 ± 0.30	82.00* ± 0.28	79.16 ± 0.29
SCORE PedsQL/School Functioning	72.82 ± 0.30	74.43* ± 0.35	75.00* ± 0.28

Notes: Values marked with *Denotes statistically significant difference (p < 0.05 for all changes from baseline). The overall quality of life score is shown in bold and the score for each subdomain of the quality of life measure is shown. Only scores for patients initiating treatment are reported here.

Abbreviation: SD, standard deviation.

Table 5 Change from Baseline to 6 and 12 Months in the Mean PedSQL Generic Core Scales (Global and Sub-Domains Physical, Emotional, Social, School Functioning), Subgroup of Patients Already Receiving Treatment

	Patients Already Undergoing Treatment
	D0	M6	M12
	n=43	n=39	n=29
SCORE TOTAL PedsQL (Mean ± SD)	77.17 ± 0.31	82.69* ± 0.26	87.17* ± 0.23
SCORE PedsQL/Physical Functioning	80.11 ± 0.31	80.44 ± 0.29	89.76* ± 0.20
SCORE PedsQL/Emotional Functioning	69.54 ± 0.29	77.3* ± 0.26	78.27* ± 0.27
SCORE PedsQL/Social Functioning	82.27* ± 0.30	91.41* ± 0.18	91.89* ± 0.19
SCORE PedsQL/School Functioning	74.88* ± 0.31	82.97* ± 0.24	87.23* ± 0.23

Notes: Values marked with *Denotes statistically significant difference (p < 0.05 for all changes from baseline). The overall quality of life score is shown in bold and the score for each subdomain of the quality of life measure is shown. Only scores for patients already undergoing treatment are reported here.

Abbreviation: SD, standard deviation.

Figure 2 Mean change from baseline in PedSQL Generic Core Scales summary scores (Global and sub-domains Physical, Emotional, Social, School functioning) after 6 and 12 months aXess supports. *p < 0.05 for all changes from baseline.

Figure 3 Percentage of response to each PedSQL questionnaire item provided by XLH patients supported by aXess support program at DO, M6 and M12 - subgroups of patients initiating treatment and patients already receiving treatment.

Table 6 Perception of the Usefulness of the Patient Support Program After 6 Months aXess Monitoring

Level of global satisfaction (0 = not at all satisfied and 10 = very satisfied)	Mean ± SD	n
	9.8 ± 0.6	45
Support in taking treatment (0 = I completely disagree / 10 = I completely agree)	Mean ± SD	n
	8.9 ± 0.6	41
Feels less anxious thanks to aXess	Disagree	Neutral	Agree	n
	6%	34%	60%	45
Frequency of follow-up calls	Suitable	Too often	% Satisfaction	n
	44	1	98%	45

Note: Satisfaction questionnaires from XLH patients after 6 months aXess monitoring.

Abbreviation: SD, standard deviation.

Table 7 Perception of the Usefulness of the Patient Support Program After 12 Months aXess Monitoring

Level of global satisfaction (0 = not at all satisfied and 10 = very satisfied)	Mean ± SD	n
	9.2 ± 1.5	34
Support in taking treatment (0 = I completely disagree / 10 = I completely agree)	Mean ± SD	n
	9.1 ± 1.8	27
Feels less anxious thanks to aXess	Disagree	Neutral	Agree	n
	0%	48%	52%	33
Frequency of follow-up calls	Suitable	Not often enough	% Satisfaction	n
	34	1	97%	35

Note: Satisfaction questionnaires from XLH patients after 12 months aXess monitoring.

Abbreviation: SD, standard deviation.