Abstract Number: PB0967
Meeting: ISTH 2020 Congress
Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Clinical
Background: Prophylaxis is recommended for severe men with Hemophilia B (MWHB) but it is unknown how widely it is adopted or what influences its use. The Community Counts Registry (Registry) provides a unique opportunity to evaluate current trends in prophylaxis usage among MWHB.
Aims: To examine factors associated with a prophylactic treatment regimen. We also considered whether treatment regimen varied according to the Hemophilia Treatment Center (HTC) where care was received.
Methods: We used marginal models to examine demographic and clinical factors associated with prophylactic compared to episodic treatment regimen among MWHB enrolled in the Registry between 2014 to 2018. Parameters were estimated with generalized estimating equations to adjust the standard errors, to account for correlated data within HTCs.
Results: Of 2,090 MWHB, 37.4% were using prophylaxis. The proportion of severe MWHB on prophylaxis varied significantly by HTC (Figure 1) and the intraclass correlation was 10.0%. In the multivariable analyses, the following were significantly associated with a prophylaxis regimen: history of a joint bleed (aOR 3.7), Hispanic ethnicity
(aOR 1.7), and no insurance (aOR 0.4) (Table 1). We observed interaction effects between disease severity and chronic pain in the previous year and between enrollment age and history of central venous access device (CVAD). Severe MWHB with chronic pain in the previous year had higher odds (aOR 14.4) then non-severe MHWB with no chronic pain. MWHB ages 4 to12 years with a history of CVAD usage had higher odds (aOR 34.9) then ages 46 years or older with no history of CVAD usage.
Conclusions: A history of a joint bleed, health insurance, ethnicity, the interaction between enrollment age and history of CVAD, and between severity and chronic pain in the previous year, were significantly associated with use of a prophylactic regimen. Additionally, we provide strong evidence that treatment regimens for MWHB vary depending on the HTC.
[Proportion of Men with Severe Hemophilia B Using a Prophylaxis Treatment Regimen in the Registry by HTC.]
Characteristics | aOR (95% CI) | p-value | Characteristics | aOR (95% CI) | p-value | |
Ethnicity: Hispanic/Latino/Spanish Ethnicity | 1.7 (1.2 – 2.5) | 0.006 | Disease Severity & Chronic Pain in past 12 months: Severe disease and Chronic Pain (yes) | 14.4 (9.2 – 22.3) | 0.0014 | |
Not Hispanic/Latino/Spanish ethnicity | reference | reference | Non-Severe disease and absent Chronic Pain | reference | reference | |
History of Joint Bleed Event | 3.7 (2.6 – 5.4) | <0.0001 | Enrollment Age and History of CVAD usage: <3 years and CVAD history (yes) | 22.0 (5.5 – 88.2) | 0.0014 | |
No History of Joint Bleed Event | reference | reference | 4-12 years and CVAD history (yes) | 34.9 (14.4 – 84.9) | 0.0002 | |
Health Insurance: Government | 1.0 (0.7 – 1.3) | 0.9606 | 13- 20 years and CVAD history (yes) | 18.1 (7.6 – 43.4) | 0.016 | |
No health insurance | 0.4 (0.2 – 0.9) | 0.0262 | 21 – 30 years and CVAD history (yes) | 6.9 (2.6 – 18.6) | 0.3729 | |
Other health insurance | 1.3 (0.6 – 2.7) | 0.4646 | 31 – 45 years and CVAD history (yes) | 1.0 (0.4 – 3.0) | 0.0806 | |
Commercial health insurance | reference | reference | >=46 years and no CVAD history | reference | reference |
[Multivariable Factors Associated with a Prophylaxis Treatment Regimen using GEE Marginal Model]
To cite this abstract in AMA style:
Bethea F, Kempton C, Hayat M. Evaluation of Factors Associated with Prophylaxis Treatment Regimen among Men with Hemophilia B Participating in the Community Counts Registry from 2014-2018 [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/evaluation-of-factors-associated-with-prophylaxis-treatment-regimen-among-men-with-hemophilia-b-participating-in-the-community-counts-registry-from-2014-2018/. Accessed March 21, 2024.« Back to ISTH 2020 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/evaluation-of-factors-associated-with-prophylaxis-treatment-regimen-among-men-with-hemophilia-b-participating-in-the-community-counts-registry-from-2014-2018/