Abstract Number: OC 09.3
Meeting: ISTH 2020 Congress
Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Clinical
Background: HA, a congenital bleeding disorder caused by a deficiency in clotting factor (F)VIII, is characterized by uncontrolled bleeding and musculoskeletal dysfunction. Little research has been conducted on the clinical burden across disease severity, especially in mild and moderate HA.
Aims: To examine clinical outcomes by disease severity in adult persons with HA (PwHA) without current FVIII inhibitors.
Methods: CHESS II (Cost of Haemophilia in Europe: a Socioeconomic Survey-II) is a retrospective, burden-of-illness study in adults with mild, moderate, and severe hemophilia (defined by endogenous FVIII/IX [IU/dL]) across 8 European countries. Data on clinical outcomes and healthcare resource utilization were captured via electronic case record forms disseminated to hemophilia specialists. Overall, 12 months’ retrospective data on bleeding episodes and joint outcomes were examined. Informed consent was obtained and the study was approved by the University of Chester ethical committee.
Results: Bleed data were available for 580/601 PwHA without current FVIII inhibitors. Mean age and body mass index were similar across severity groups (Table 1). Only 9% and 2% of persons with moderate and mild HA, respectively, received prophylaxis. In moderate HA, mean annual bleed rate (ABR) was 3.06 (standard deviation [SD], 7.5); marginally different to in severe HA (mean [SD], 3.91 [6.4]). In moderate HA, 15% had zero bleeds in the past year, 39% had problem joints (i.e., chronically damaged joints), and 24% had joint surgery. In mild HA, mean ABR (SD) was 2.12 (10.1). Notably, only 26% of persons with mild HA had zero bleeds in the past year, 17% had problem joints and 7% had joint surgery, indicating a potential unmet need in this population (Table 2).
Conclusions: Persons with mild and moderate HA appear to exhibit a clinical burden and potential unmet medical need expressed in bleeding frequency, problem joints and incidence of surgeries to affected joints.
| Mild HA (n=97) |
Moderate HA (n=199) |
Severe HA (n=284) |
All (N=580) |
|
| Mean age (SD), years | 39.41 (14.8) | 38.82 (15.1) | 37.24 (13.8) | 38.15 (14.4) |
| Mean BMI (SD) | 24.59 (2.4) | 24.59 (3.0) | 24.65 (2.8) | 24.62 (2.8) |
| Treatment strategy, n (%) | ||||
| No treatment | 69 (71) | 120 (60) | 0 | 189 (33) |
| Primary on-demand* | 26 (27) | 50 (25) | 105 (37) | 181 (31) |
| Primary prophylaxis | 0 | 0 | 28 (10) | 28 (5) |
| Secondary on-demand | 0 | 12 (6) | 23 (8) | 35 (6) |
| Secondary prophylaxis§ | 2 (2) | 17 (9) | 128 (45) | 147 (25) |
| *Primary on-demand: treatment has always been episodic or ‘on-demand’. Primary prophylaxis: treatment has always been prophylactic. Secondary on-demand: previously on prophylaxis, currently receiving treatment episodically or ‘on-demand’. §Secondary prophylaxis: treatment was previously episodic or ‘on-demand’, currently on prophylaxis. BMI, body mass index; HA, hemophilia A; SD, standard deviation. |
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[Table 1. Baseline Demographics and Characteristics]
| Mild HA (n=97) | Moderate HA (n=199) | Severe HA (n=284) | All (N=580) | |
| Mean annual bleed rate (SD) | 2.12 (10.1) | 3.06 (7.5) | 3.91 (6.4) | 3.32 (7.5) |
| Zero bleeds, n (%) | 25 (26) | 29 (15) | 21 (8) | 75 (13) |
| 1 bleed, n (%) | 44 (45) | 57 (29) | 51 (18) | 152 (26) |
| ≥2 bleeds, n (%) | 28 (29) | 113 (57) | 212 (75) | 353 (61) |
| Target joint, n (%) | 8 (8) | 46 (23) | 128 (45) | 182 (31) |
| Problem joint, n (%) | 17 (17) | 77 (39) | 138 (49) | 232 (40) |
| Joint surgery*, n (%) | 7 (7) | 48 (24) | 73 (26) | 128 (22) |
| *Target joint surgery includes: arthrocentesis (joint aspiration), arthrodesis (joint fusion), arthroplasty (joint reconstruction/replacement), arthroscopy (joint examination/repair via endoscope), and synovectomy (removal of synovium). HA, hemophilia A; SD, standard deviation. |
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[Table 2. The Frequency of Bleeds and Joint Damage in Persons with Mild, Moderate, and Severe HA]
To cite this abstract in AMA style:
Nissen F, Burke T, Asghar S, Ferri Grazzi E, Shang A, Castro F, Aizenas M, Meier O, O'Hara J. An Insight into Clinical Outcomes in Mild, Moderate, and Severe Hemophilia A (HA): A Preliminary Analysis of the CHESS II Study [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/an-insight-into-clinical-outcomes-in-mild-moderate-and-severe-hemophilia-a-ha-a-preliminary-analysis-of-the-chess-ii-study/. Accessed September 27, 2023.« Back to ISTH 2020 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/an-insight-into-clinical-outcomes-in-mild-moderate-and-severe-hemophilia-a-ha-a-preliminary-analysis-of-the-chess-ii-study/