Abstract Number: PB1120
Meeting: ISTH 2020 Congress
Theme: Hemophilia and Rare Bleeding Disorders » Management of Bleeding and Trauma
Background: During the past decades, hemophilia A (HA) treatment has evolved tremendously, with a significant decrease in treatment-associated risks and side effects.
Aims: To investigate medical/orthopedic/surgical treatment options in Romanian severe HA patients.
Methods: Retrospective analysis of the hospitalization charts of severe HA patients, diagnosed in the Hematology Department, Fundeni Clinical Institute, Bucharest, Romania. All patients agreeing to partake in the study signed an informed consent and were tested for the presence of the intron 22 inversion (inv22) by inverse-shifting polymerase chain reaction. The approval of the Ethics Council was granted (approval number 4203/31.01.2018). We respected the regulations imposed by the national law and Helsinki Declaration (1975, revised in 5/2008).
Results: The study group involved 33 patients with severe HA (mean age=40.64±14.92 years): 48.48% (n=16) < 35 years and 51.52% (n=17) ≥35 years. All patients received FVIII substitution and 60.61% (n=20) received intermittent FVIII prophylaxis (x3 times/week), the latter more likely employed in patients < 35 years vs. ≥35 years (81.25% vs. 41.18%, P=0.0324). Eptacog alpha (recombinant FVIIa) was prescribed in 3 patients who developed inhibitors (9.09%). There were no significant differences in terms of inhibitor development among inv22-positive vs. inv22-negative or < 35 years vs. ≥35 years (P=0.2273). Patients with arthropathy required synovectomy in 6.06% (n=2) of cases and total arthroplasty in 15.15% (n=5) of cases. Physiotherapy was employed in 48.48% (n=16) of severe HA patients, particularly in inv22-positive vs. inv22-negative cases (75.00% vs. 33.33%, P=0.0299). Detailed results are depicted in Table 1 and Table 2.
Conclusions: Management of severe HA is complex, requiring a collaborative effort between specialists in hematology, orthopedics and physical&rehabilitation medicine. In our study, inv22-positivity was linked to a higher need for physiotherapy, probably due to the severity of bleeding episodes. Patients ≥35 had higher needs for intermittent FVIII prophylaxis.
Funding: Research.Grant.Competition.For.Students-SSMB-contract-no.-231/29.03.2017.
[Treatment Differences between Hemophilia A Patients Positive and Negative for the Intron 22 Inversion.]
[Treatment Differences between Hemophilia A Patients <35 and ≥35 Years Old.]
To cite this abstract in AMA style:
Gaman M-, Brinza M, Uscatescu V, Diaconu A, Grigore A, Dragomir M, Coriu D. Treatment Options in Severe Hemophilia A Patients from Romania: What Role did the Intron 22 Inversion and Age Play? [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/treatment-options-in-severe-hemophilia-a-patients-from-romania-what-role-did-the-intron-22-inversion-and-age-play/. Accessed March 21, 2024.« Back to ISTH 2020 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/treatment-options-in-severe-hemophilia-a-patients-from-romania-what-role-did-the-intron-22-inversion-and-age-play/