Direct and indirect costs and cost-driving factors of Tuberous sclerosis complex in children, adolescents, and caregivers: a multicenter cohort study

Grau, Janina; Zöllner, Johann Philipp; Schubert-Bast, Susanne; Kurlemann, Gerhard; Hertzberg, Christoph; Wiemer-Kruel, Adelheid; Bast, Thomas; Bertsche, Astrid; Bettendorf, Ulrich; Fiedler, Barbara; Hahn, Andreas; Hartmann, Hans; Hornemann, Frauke; Immisch, Ilka; Jacobs, Julia; Kieslich, Matthias; Klein, Karl Martin; Klotz, Kerstin A.; Kluger, Gerhard; Knuf, Markus; Mayer, Thomas; Marquard, Klaus; Meyer, Sascha; Muhle, Hiltrud; Müller-Schlüter, Karen; Noda, Anna H.; Ruf, Susanne; Sauter, Matthias; Schlump, Jan-Ulrich; Syrbe, Steffen; Thiels, Charlotte; Trollmann, Regina; Wilken, Bernd; Willems, Laurent M.; Rosenow, Felix; Strzelczyk, Adam

doi:10.1186/s13023-021-01899-x

Orphanet Journal of Rare Diseases

Table 6 Univariate and multivariate analysis of cost-driving factors for total direct, total indirect and nursing care level costs (3-months period in 2019 Euro)

From: Direct and indirect costs and cost-driving factors of Tuberous sclerosis complex in children, adolescents, and caregivers: a multicenter cohort study

	n	Total direct costs in €	Median	SD	p value^§	Total indirect costs in €	Median	SD	p value^§	Nursing care level costs in €	Median	SD	p value^§
Gender					0.207				0.839				0.809
Male	95	5213	2655	5927		3291	1371	4063		1149	948	1002
Female	89	4667	1873	6259		3071	548	4612		1178	1635	1032
Age					0.632*				0.099*				0.005*
0 to 3 years	24	4554	2786	5358		4396	3231	4771		589	474	645
4 to 10 years	78	5047	2519	5970		2923	269	4635		1127	948	1027
11 to < 22 years	73	5088	1556	6578		2796	645	3687		1358	1635	1042
Number of antiseizure drugs					< 0.001^#				0.002				< 0.001
≥ 2	94	6829	3205	7147		4172	2553	4814		1458	1635	956
0–1	90	2985	1256	3871		2153	215	3488		855	0	985
mTOR inhibitors intake					< 0.001^#				0.087				0.097
Yes	49	11,459	11,358	5945		2322	430	3795		1367	1635	1023
No	135	2586	1346	4085		3497	1371	4476		1089	948	1004
Seizures					< 0.001				0.006				0.002
Recurrent seizures	88	6852	3765	6821		4014	2258	4768		1408	1635	992
Seizure free > 1 year or no seizures	96	3204	1196	4707		2423	215	3744		938	948	984
Epilepsy					0.002				0.043				< 0.001
Yes (91.8%)	169	5230	2384	6213		3351	860	4390		1251	1635	1003
No (8.2%)	15	1778	711	2839		1308	0	3051		172	0	473
Structural brain disorders					0.022				0.059				0.170
Yes (83.2%)	153	5321	2388	6293		3369	860	4424		1211	1635	993
No (16.8%)	31	3111	1211	4541		2273	0	3742		925	0	1096
Psychiatric disorders					< 0.001				< 0.001^#				< 0.001^#
Yes (51.1%)	94	6372	2802	6564		4387	2848	4883		1727	2184	890
No (48.9%)	90	3463	1213	5157		1929	161	3233		574	0	774
Heart and circulatory manifestations					0.008				0.133				0.059
Yes (66.8%)	123	5755	2655	6678		3550	1097	4394		1257	1635	1044
No (33.2%)	61	3324	1611	4245		2447	430	4125		973	948	930
Kidney and urinary tract manifestations					0.392				0.113				0.010
Yes (53.3%)	98	4714	1993	6227		3552	1075	4380		1341	1635	1028
No (46.7%)	86	5217	2389	5931		2765	269	4252		960	948	964
Skin manifestations					0.150				0.659				0.110
Yes (85.9%)	158	4676	1890	6106		3139	645	4206		1212	1635	1018
No (14.1%)	26	6606	4946	5749		3457	323	5082		865	474	950
Other disorders					0.600				0.899				0.991
Yes (29.9%)	55	4134	2000	4685		2868	548	3808		1166	948	982
No (70.1%)	129	5296	2122	6570		3319	645	4537		1162	1635	1031
Level of disability					< 0.001				< 0.001^#				< 0.001^#
None or ≤ 60%	61	3515	1057	5560		1544	0	3235		187	0	529
70–100%	123	5660	2495	6221		3998	2742	4574		1647	1635	831
Total Disorders					0.009*				0.016*				< 0.001*
1–2 Manifestations (8.7%)	16	1282	894	1365		1593	0	3805		323	0	604
3 Manifestations (13%)	24	3705	1522	4194		1637	0	2811		403	0	736
4 Manifestations (17.9%)	33	5916	2085	6097		3475	1468	5171		1139	948	900
5 Manifestations (32.6%)	60	5839	2432	7021		3087	860	4037		1367	1635	972
6–7 Manifestations (27.8%)	51	5011	2403	6168		4337	2957	4561		1560	1635	1027

^§Mann–Whitney-U-test
*Kruskal–Wallis-test; SD = standard deviation
^#Significant predictor in multivariate analysis after Bonferroni correction

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ISSN: 1750-1172

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