Background

The ability of a person with hemophilia to adhere to treatment recommendations aimed at preventing and controlling bleeding likely impacts clinical outcomes and resource utilization. Annual cost of replacement factor for persons with severe hemophilia on prophylaxis is estimated to be as high as $300,000 per year (Johnson KA and Zhou ZY. ASH Annual Meeting Educational Program. 2011; 413-418). Hemophilia care is optimized when delivered by a highly specialized hemophilia treatment center (HTC) (Soucie et al. Blood. 2000; 96: 437-442). Consolidation of care for persons with hemophilia from a large geographic region within an HTC results in demographic heterogeneity that may impact an individual's ability to adhere to treatment recommendations. Little is known regarding the impact of socio-demographic factors on treatment outcomes for persons with hemophilia. The purpose of this study was to identify disease-related, treatment-related and demographic variables that have a significant impact on morbidity and resource utilization.

Methods

We identified 69 persons with severe hemophilia treated at our HTC between June 2009 and June 2012. We collected data for the variables listed below in table 1. To assess morbidity and resource utilization, we collected data for total factor use, total outpatient cost, total inpatient cost, frequency of inpatient and outpatient encounters and length of stay when hospitalized. Risk factors were identified using linear regression. A subset analysis was performed to evaluate the impact of prophylaxis on patients without inhibitors. All statistical analyses were performed using STATA (version 12.0 College Station, TX). Statistical significance was defined as P < 0.05.

Results

Age and inhibitor status were the only variables to significantly impact cost in both inpatient and outpatient settings (Table 1). The statistically significant beneficial impact of prophylaxis was confirmed by subgroup analysis of patients without inhibitors with respect to number of hospitalizations and length of stay, but not total cost.

Table 1

Total Cost

Total Outpatient CostTotal Inpatient Cost
VariableCoefficientP-valueCoefficientP-value
Age     
0-18 (Reference) $33,098.70  $4,304.96  
19-24 $41,229.18 0.569 $1,206,743.00 0.043 
25-39 $6,908.62 0.910 $111,033.10 0.824 
40+ $177,164.30 0.006 $561,069.60 0.274 
Race     
White (Reference) $84,293.73  $159,972.60  
Non-White -$27,089.41 0.669 $886,072.10 0.074 
Level of Education     
High School (Reference) $103,974.60  $363,663.60  
Post High School -$71,256.01 0.168 -$105,655.90 0.797 
Distance     
<20 (Reference) $76,639.79  $677,486.50  
20-39 -$34,645.45 0.628 -$536,091.80 0.342 
40-59 $17,297.19 0.806 -$339,293.80 0.540 
>60 $24,235.24 0.727 -$546,892.20 0.318 
Population Density     
<500 (Reference) $79,850.63  $222,397.60  
500-1500 $38,479.01 0.531 -$111,211.20 0.344 
>1500 -$35,152.90 0.548 $438,733.90 0.454 
     
Inhibitor     
No (Reference) $42,779.31  $12,317.91  
Yes $139,574.00 0.011 $1,205,950.00 0.005 
HIV Positive     
No (Reference) $63,518.41  $388,921.60  
Yes $90,111.22 0.165 -$356,545.60 0.490 
HCV Positive     
No (Reference) $46,779.09  $271,800.40  
Yes $93,181.17 0.070 $158,450.70 0.700 
HBV Positive     
No (Reference) $63,683.04  $236,377.60  
Yes $71,331.68 0.233 $416,466.10 0.379 
Family History     
No (Reference) $75,939.89  $79,381.42  
Yes $5,606.31 0.911 $426,993.20 0.280 
     
Home Therapy     
No (Reference) $79,683.84  $63,368.37  
Yes -$540.95 0.995 $288,644.90 0.678 
Prophylaxis     
No (Reference) $136,291.60  $855,492.40  
Yes -$91,628.24 0.070 -$848,184.50 0.033 
Product     
Plasma Derived (Reference) $126,870.50  $31,583.86  
Recombinant -$98,638.07 0.135 -$24,181.53 0.962 
Bypass $193,397.80 0.026 $2,401,224.00 0.001 
Primary Caregiver     
No (Reference) $115,912.00  $560,892.00  
Yes -$87,373.20 0.078 -$556,707.00 0.158 
Total Outpatient CostTotal Inpatient Cost
VariableCoefficientP-valueCoefficientP-value
Age     
0-18 (Reference) $33,098.70  $4,304.96  
19-24 $41,229.18 0.569 $1,206,743.00 0.043 
25-39 $6,908.62 0.910 $111,033.10 0.824 
40+ $177,164.30 0.006 $561,069.60 0.274 
Race     
White (Reference) $84,293.73  $159,972.60  
Non-White -$27,089.41 0.669 $886,072.10 0.074 
Level of Education     
High School (Reference) $103,974.60  $363,663.60  
Post High School -$71,256.01 0.168 -$105,655.90 0.797 
Distance     
<20 (Reference) $76,639.79  $677,486.50  
20-39 -$34,645.45 0.628 -$536,091.80 0.342 
40-59 $17,297.19 0.806 -$339,293.80 0.540 
>60 $24,235.24 0.727 -$546,892.20 0.318 
Population Density     
<500 (Reference) $79,850.63  $222,397.60  
500-1500 $38,479.01 0.531 -$111,211.20 0.344 
>1500 -$35,152.90 0.548 $438,733.90 0.454 
     
Inhibitor     
No (Reference) $42,779.31  $12,317.91  
Yes $139,574.00 0.011 $1,205,950.00 0.005 
HIV Positive     
No (Reference) $63,518.41  $388,921.60  
Yes $90,111.22 0.165 -$356,545.60 0.490 
HCV Positive     
No (Reference) $46,779.09  $271,800.40  
Yes $93,181.17 0.070 $158,450.70 0.700 
HBV Positive     
No (Reference) $63,683.04  $236,377.60  
Yes $71,331.68 0.233 $416,466.10 0.379 
Family History     
No (Reference) $75,939.89  $79,381.42  
Yes $5,606.31 0.911 $426,993.20 0.280 
     
Home Therapy     
No (Reference) $79,683.84  $63,368.37  
Yes -$540.95 0.995 $288,644.90 0.678 
Prophylaxis     
No (Reference) $136,291.60  $855,492.40  
Yes -$91,628.24 0.070 -$848,184.50 0.033 
Product     
Plasma Derived (Reference) $126,870.50  $31,583.86  
Recombinant -$98,638.07 0.135 -$24,181.53 0.962 
Bypass $193,397.80 0.026 $2,401,224.00 0.001 
Primary Caregiver     
No (Reference) $115,912.00  $560,892.00  
Yes -$87,373.20 0.078 -$556,707.00 0.158 
Conclusions

Identifying and addressing potential barriers to care will likely reduce morbidity and cost for persons with hemophilia. Caregiver status and age are demographic variables that may contribute to poor medical adherence resulting in increased morbidity and cost of care for persons with severe hemophilia.

Disclosures:

No relevant conflicts of interest to declare.

Author notes

*

Asterisk with author names denotes non-ASH members.

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