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3986 Descriptive and Epidemiological Study of Joint Damage in Non-Severe Hemophilia: Update of the Damage Study of the Mediterranean GroupClinically Relevant Abstract

Program: Oral and Poster Abstracts
Session: 322. Disorders of Coagulation or Fibrinolysis: Clinical and Epidemiological: Poster III
Hematology Disease Topics & Pathways:
adult, pediatric, Study Population, Human
Monday, December 11, 2023, 6:00 PM-8:00 PM

Ana Marco Rico, MD, PhD1*, Jose Manuel Calvo-Villas, MD2*, Francisco Jose Lopez Jaime, MD3*, Mariana Canaro Hinryk, MD4*, Sonia Herrero-Martín, MD, PhD5*, Laura Entrena Ureña, MD6*, Shally Marcellini Antonio, MD7*, Maria del Mar Nieto, MD8*, Bolivar Diaz Jordan, MD9*, Covadonga Garcia Diaz, MD10*, Sergio Jurado Herrera, MD11*, Faustino García Candel, MD, PhD12*, Miguel Blanquer Blanquer, MD, PhD13, Pascual Marco VERA, MD, PhD14,15* and Ihosvany Fernandez-Bello, BSPharm, PhD16,17*

1Thrombosis and Hemostasis Unit, Hospital General Universitario Dr. Balmis de Alicante-ISABIAL, Alicante, Spain
2Hospital Universitario Miguel Servet, Zaragoza, Spain
3Thrombosis and Hemostasis Unit, Hospital Universitario Regional de Málaga-IBIMA, Malaga, Spain
4Hospital Universitario Son Espases, Palma de Mallorca, Spain
5Hospital Universitario de Guadalajara, Guadalajara, Spain
6Hospital Universitario Virgen de las Nieves, Granada, Spain
7Complejo Asistencial de Segovia, Segovia, Spain
8Complejo Hospitalario de Jaén, Jaen, Spain
9Hospital General de Valdepeñas, Valdepeñas, Spain
10Hospital Universitario de Burgos, Burgos, Spain
11Hospital Universitario Torrecárdenas, Almeria, Spain
12Hospital Clínico Universitario Virgen de la Arrixaca, Murcia, Spain
13Hospital Clínico Universitario Virgen De La Arrixaca, Murcia, Spain
14Hospital General Universitario Dr. Balmis de Alicante-ISABIAL, Alicante, Spain
15Department of Clinical Medicine, Universidad Miguel Hernández de Elche, Elche, Spain
16Thrombosis and Hemostasis Unit, Hospital General Universitario Dr. Balmis-ISABIAL, Alicante, Spain
17Thrombosis and Hemostasis Unit, Hospital Universitario Regional De Málaga-IBIMA, Málaga, Spain

Introduction: There is growing evidence indicating joint damage (JD) in non-severe hemophilia (nSH) patients. Although age and baseline factor (F) VIII/FIX levels have been proposed as good predictors of JD in mild hemophilia (MiH), data are scarce and in moderate hemophilia (MoH) are lacking. Besides, global hemostatic capacity (GHC) in this population and its contribution to JD has not been assessed.

Methods: We designed a cross-sectional, observational and multicenter study carried out in 12 Spanish hospitals (Mediterranean Group). We evaluated JD in nSH patients and its correlation with age, baseline FVIII/FIX and GHC. JD was defined as a HEAD-US score >0. Baseline FVIII (chromogenic assay), FIX (clotting assay) and GHC (assessed by thrombin generation test, TGT, Genesia®, Stago) were evaluated in a central laboratory. Results were expressed as median (25th-75th percentiles). Categorical variables were expressed with percentage. A p value<0.05 was considered statistically significant.

Results: A total of 98 nSH patients were included: 10 (10.2%) MoH (9 HA: age= 35.6 [21.7-45.0] years old (YO); FVIII = 4.0 [4.0-5.0] IU/dL; 1 HB: FIX= 2 IU/dL) and 88 MiH (84 HA: age= 38.6 [19.0-52.1 YO]; FVIII= 15.5 [11.0-24.0] IU/dL; 4 HB: FIX= 30.0 [16.8-35.4] IU/dL). Target joints were observed in patients with MiH (7/88: 8.6% of the MiH patients). HEAD-US score was obtained in 73 patients (74.5% of the total participating patients): nine MoH patients presented a HEAD-US= 1 (0-6) (age: 32.7 [21.7-42.9] YO) while 64 MiH patients presented a HEAD-US= 0 (0-3) (age: 37.9 [17.8-48.7] YO) (comparison between MoH and MiH patients: HEAD-US: p=0.427, age: p= 0.614). Only 55.5% (5/9) MoH patients and 45.3% (29/64) MiH patients, presented JD (p=0.725). Baseline FVIII/FIX was 13.0 (7.0-20.0) IU/dL and 16.0 (11.0-22.5) IU/dL in patients with or without JD respectively (p= 0.311). These mild difference of number of patients with JD and degree of JD between MiH and MoH were corroborated by not detecting correlation between baseline FVIII/FIX levels and JD (r= -0.095; p= 0.426) (Figure 1). JD increased with age (r= 0.380; p= 0.001) reinforcing previous findings. GHC correlated with baseline FVIII/FIX (Table 1) but not with the degree of JD, suggesting that thrombin generation, with the used conditions, might not be helpful to predict JD in this population.

Conclusions: Around 50% of patients with nSH presented JD. This JD increased with age, supporting previous data. Although there was a correlation between baseline FVIII/FIX levels and thrombin generation, none of the TGT parameters were predictive of the degree of JD, enhancing that other variables might be influencing the development of JD in nSH patients. Of note, MiH individuals may develop severe JD due to presence of target joints. In summary, our results highlight the importance of evaluating JD in nSH in order to determine whether new protocols are required for the diagnosis, prevention, and treatment of JD in nSH patients.

Disclosures: No relevant conflicts of interest to declare.

*signifies non-member of ASH