Healthcare costs and resource utilization in patients with severe aplastic anemia in the US

Figures & data

Figure 1. Study schematic.

Figure 2. Patient selection.

Table 1. Demographic characteristics.

	Age group					p-value^a
	Overall (n = 939)	2–17 years (n = 101)	18–44 years (n = 161)	45–64 years (n = 358)	≥65 years (n = 319)
Age, years, mean (SD)	54.0 (23.3)	9.7 (4.8)	30.3 (8.4)	56.2 (5.2)	77.6 (7.3)
Sex, n (%)
Male	463 (49.3)	58 (57.4)	83 (51.6)	158 (44.1)	164 (51.4)	0.0606
CCI at baseline, mean (SD)	3.57 (3.32)	1.88 (2.21)	2.48 (3.09)	3.97 (3.39)	4.21 (3.34)	<0.0001
Comorbidities, n (%)
Type 2 diabetes without chronic complications	205 (21.8)	2 (2.0)	12 (7.5)	80 (22.3)	111 (34.8)
Chronic pulmonary disease	180 (19.2)	14 (13.9)	22 (13.7)	66 (18.4)	78 (24.5)
Renal disease	158 (16.8)	5 (5.0)	10 (6.2)	52 (14.5)	91 (28.5)
Metastatic solid tumor	149 (15.9)	7 (6.9)	18 (11.2)	80 (22.3)	44 (13.8)
Mild liver disease	126 (13.4)	9 (8.9)	23 (14.3)	53 (14.8)	41 (12.9)
Congestive heart failure	121 (12.9)	3 (3.0)	13 (8.1)	34 (9.5)	71 (22.3)
Peripheral vascular disease	98 (10.4)	3 (3.0)	6 (3.7)	27 (7.5)	62 (19.4)
Cerebral vascular disease	79 (8.4)	7 (6.9)	7 (4.3)	25 (7.0)	40 (12.5)
Type 2 diabetes with chronic complications	78 (8.3)	0 (0.00)	3 (1.9)	22 (6.1)	53 (16.6)
Rheumatic disease	44 (4.7)	1 (1.0)	8 (5.0)	20 (5.6)	15 (4.7)
Myocardial infarction	27 (2.9)	0 (0)	4 (2.5)	8 (2.2)	15 (4.7)
Moderate-to-severe liver disease	21 (2.2)	3 (3.0)	4 (2.5)	11 (3.1)	3 (0.9)
Peptic ulcer disease	21 (2.2)	0 (0)	1 (0.6)	6 (1.7)	14 (4.4)
Dementia	8 (0.9)	0 (0.00)	0 (0.00)	0 (0.00)	8 (2.5)
Hemiplegia or paraplegia	7 (0.7)	1 (1.0)	3 (1.9)	1 (0.3)	2 (0.6)
AIDS/HIV	6 (0.6)	0 (0.00)	4 (2.5)	1 (0.3)	1 (0.3)
US region, n (%)
Northeast	205 (21.8)	24 (23.8)	24 (14.9)	67 (18.7)	90 (28.2)	0.0000
North Central	226 (24.1)	20 (19.8)	32 (19.9)	70 (19.6)	104 (32.6)
South	349 (37.2)	38 (37.6)	72 (44.7)	162 (45.3)	77 (24.1)
West	156 (16.6)	19 (18.8)	33 (20.5)	58 (16.2)	46 (14.4)
Unknown	3 (0.3)	0 (0)	0 (0)	1 (0.3)	2 (0.6)
Health insurance, n (%)
FFS	819 (87.2)	96 (95.0)	143 (88.8)	306 (85.5)	274 (85.9)	0.0445
HMO and POS capitation	100 (10.6)	5 (5.0)	15 (9.3)	39 (10.9)	41 (12.9)
Other	20 (2.1)	0 (0)	3 (1.9)	13 (3.6)	4 (1.3)

^a p-values represent comparison across age groups.

Abbreviations. AIDS, acquired immune deficiency syndrome; CCI, Charlson Comorbidity Index; FFS, fee for service; HIV, human immunodeficiency virus infection; HMO, Health Maintenance Organization; LOS, length of stay (hospital); n, total number within a sub-category; POS, point of service; SD, standard deviation; US, United States.

Table 2. First-line treatments.

Treatment	n (%) (n = 939)
BMS	188 (20.0)
BMS plus steroid/prednisone	171 (18.2)
Calcineurin plus steroid/prednisone	76 (8.1)
Calcineurin	75 (8.0)
BMS plus cyclophosphamide and steroid/prednisone	66 (7.0)
Blood transfusions	52 (5.5)
Calcineurin plus EPAG and steroid/prednisone	23 (2.4)
Cyclophosphamide plus steroid/prednisone	22 (2.3)
Calcineurin plus HSCT	21 (2.2)
Cyclophosphamide	21 (2.2)
Steroid/prednisone	21 (2.2)
BMS plus calcineurin and steroid/prednisone	20 (2.1)
BMS plus calcineurin and HSCT	17 (1.8)
Androgens	16 (1.7)
Calcineurin plus HSCT and steroid/prednisone	12 (1.3)
BMS plus calcineurin and HSCT and steroid/prednisone	11 (1.2)
Androgens plus steroid/prednisone	10 (1.1)
BMS plus calcineurin	9 (1.0)
EPAG	9 (1.0)
Other treatments*	97 (10.3)

* Representative of various treatment regimens being used during the first 6 months, each with proportion <1%.

Abbreviations. BMS, bone marrow stimulants; EPAG, Eltrombopag; HSCT, hematopoietic stem cell transplantation.

Table 3. Monthly all-cause and SAA-related HCRU in the follow-up period.

	Age group					p-value^c
	Overall^a(n = 939)	2–17 years (n = 101)	18–44 years (n = 161)	45–64 years (n = 358)	≥65 years (n = 319)
Monthly all-cause HCRU, mean (SD)
LOS (in days)	1.65 (2.61)	2.47 (2.78)	1.88 (3.07)	1.87 (2.91)	1.03 (1.64)	<0.0001
No of Hospitalizations	0.18 (0.23)	0.31 (0.39)	0.18 (0.21)	0.20 (0.22)	0.13 (0.15)	<0.0001
No of ER visits	0.25 (0.30)	0.25 (0.33)	0.19 (0.26)	0.25 (0.30)	0.30 (0.30)	<0.0001
No of office visits	2.24 (1.40)	2.62 (1.61)	2.18 (1.43)	2.27 (1.50)	2.12 (1.18)	0.0461
No of outpatient visits^b	2.90 (2.64)	3.53 (2.02)	2.85 (2.43)	3.03 (3.12)	2.59 (2.26)	<0.0001
Monthly SAA-related HCRU, mean (SD)
LOS (in days)	0.18 (0.70)	0.56 (1.30)	0.35 (0.83)	0.11 (0.59)	0.04 (0.28)	<0.0001
No of hospitalizations	0.01 (0.04)	0.03 (0.05)	0.02 (0.05)	0.01 (0.03)	0.01 (0.03)	<0.0001
No of ER visits	0.02 (0.07)	0.06 (0.16)	0.02 (0.06)	0.01 (0.04)	0.02 (0.05)	<0.0001
No of office visits	0.46 (0.99)	1.16 (1.63)	0.88 (1.30)	0.27 (0.64)	0.24 (0.67)	<0.0001
No of outpatient visits^b	0.55 (1.31)	1.28 (1.70)	0.98 (1.67)	0.39 (1.15)	0.28 (0.95)	<0.0001
No of blood transfusions	0.15 (0.57)	0.20 (0.40)	0.25 (0.65)	0.12 (0.61)	0.11 (0.52)	<0.0001

^a Mean (SD) follow-up per patient was 21.54 (11.36) months.

^b Outpatient settings were captured as a hospital setting or ambulatory surgical center, a skilled nursing facility, a residential substance abuse center, a comprehensive outpatient rehabilitation center, an end-stage-renal disease center or an outpatient NEC center.

^c p-values represent comparison across age groups.

Abbreviations. ER, emergency room; HCRU, healthcare resource utilization; LOS, length of stay (hospital); No, number; SAA, severe aplastic anemia; SD, standard deviation.

Table 4. Monthly all-cause and SAA-related direct costs in the follow-up period.

	Age group					p-value^c
	Overall (n = 939)	2–17 years (n = 101)	18–44 years (n = 161)	45–64 years (n = 358)	≥65 years (n = 319)
Monthly all-cause direct costs, mean (SD)
Hospitalizations	$11,433 ($25,040)	$21,914 ($28,665)	$15,963 ($38,709)	$12,326 ($23,075)	$4,826 ($12,223)	<0.0001
ER visits	$624 ($1,703)	$497 ($678)	$418 ($682)	$779 ($2,418)	$594 ($1,251)	0.1888
Office visits	$528 ($694)	$823 ($785)	$546 ($544)	$551 ($849)	$399 ($472)	<0.0001
Outpatient visits^a	$7,615 ($13,273)	$8,804 ($7,765)	$8,327 ($13,208)	$7,826 ($10,317)	$6,643 ($17,079)	<0.0001
Prescription costs	$5,998 ($11,461)	$7,892 ($15,129)	$8,999 ($18,169)	$6,611 ($10,330)	$3,195 ($3,950)	0.0004
Other costs^b	$2,083 ($4,292)	$1,824 ($3,308)	$1,965 ($4,590)	$2,157 ($4,035)	$2,142 ($4,688)	0.0461
Total	$28,280 ($36,127)	$41,755 ($41,765)	$36,217 ($51,747)	$30,248 ($32,357)	$17,798 ($23,969)	<0.0001
Monthly SAA-related direct costs, mean (SD)
Hospitalizations	$1,608 ($7,774)	$5,941 ($15,778)	$2,538 ($8,945)	$1,125 ($6,529)	$309 ($1,866)	<0.0001
ER visits	$47 ($257)	$139 ($493)	$62 ($181)	$28 ($265)	$32 ($139)	<0.0001
Office visits	$127 ($374)	$423 ($771)	$233 ($415)	$53 ($136)	$65 ($274)	<0.0001
Outpatient visits^a	$1,462 ($4,994)	$3,444 ($5,529)	$2,800 ($7,935)	$845 ($3,579)	$851 ($3,863)	<0.0001
Prescription costs	$4,451 ($10,552)	$6,226 ($14,382)	$7,401 ($16,511)	$4,820 ($9,588)	$1,985 ($3,371)	0.0008
Other costs^b	$190 ($1,048)	$537 ($1,962)	$362 ($1,533)	$112 ($756)	$80 ($367)	<0.0001
Total	$7,884 ($16,254)	$16,709 ($23,922)	$13,395 ($22,100)	$6,981 ($14,744)	$3,321 ($6,905)	<0.0001

^a Outpatient settings were captured as a hospital setting or ambulatory surgical center, a skilled nursing facility, a residential substance abuse center, a comprehensive outpatient rehabilitation center, an end-stage-renal disease center, or an outpatient NEC center.

^b Other costs are expenses related to school, homeless shelters, assisted living facilities, group homes, mobile units, temporary lodging, health clinics, places of employment, military treatment facilities, custodial care facilities, adult living facilities, Federal qualified health centers, community mental health centers, intermediate care facilities, physical residential treatment centers, non-residential substance abuse centers, mass immunization centers, State or local public health centers, rural health clinics, independent laboratories, and other miscellaneous costs.

^c p-values represent comparison across age groups.

Abbreviations. ER, emergency room; SAA, severe aplastic anemia; SD, standard deviation.

Table 5. Examples of yearly economic burden of chronic hematologic diseases.

Reference	Disease	Total costs (USD) PPPY
Current study	Severe aplastic anemia	$236,220–$640,812
Dieguez et al.^14 (2018)	Acute leukemia	$463,414
	Multiple myeloma	$213,879
	Bone marrow disorders	$133,744
	Lymphoma	$130,545
	Chronic leukemia	$88,913

Abbreviations. USD, US dollars; PPPY, per person per year.

Dieguez G, Ferro C, Rotter D. Milliman Research Report. The cost burden of blood cancer care. Commissioned by the Leukemia & Lymphoma Society; 2018.

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