Adherence to prophylactic treatment in adult patients with severe hemophilia in Poland.

INTRODUCTION: Patient adherence to a prophylactic regimen is important for optimal benefit of hemophilia treatment. Despite a growing number of adults with hemophilia in Poland receiving secondary prophylaxis, data on adherence to the regimen are limited. OBJECTIVES: The aim of the study was to assess adherence to secondary prophylaxis in Polish adults with severe hemophilia. PATIENTS AND METHODS: Patients were recruited in 18 hemophilia treatment centers in Poland. Adherence to prophylaxis was assessed with the Validated Hemophilia Regimen Treatment Adherence Scale Prophylaxis (VERITAS­Pro) questionnaire. RESULTS: Data on 270 men on the prophylactic regimen (median [interquartile range, IQR] age, 37 [18-75] years; mean [SD], 38.2 [13.3] years) were analyzed. Median (IQR) VERITAS­Pro score for the study population was 36 (24-76) years; mean (SD), 37.7 (9.9) years, indicating general adherence to the prophylactic regimen. The median subscale scores ranged from 4 for Dosing to 8 for Planning (means, 5.6 and 7.7, respectively). The most pronounced difference in the subscale scores between adherent and nonadherent patients was recorded for Dosing (median, 4 vs 10; mean, 5.3 vs 9.3) and Remembering (median, 5 vs 11; mean, 5.7 vs 10.7). The overall adherence rate was 94%. CONCLUSIONS: Our results show a high rate of adherence to hemophilia prophylaxis by Polish adults. Problems with the management of clotting factor stocks and remembering about the injection of the clotting factor were identified as potential barriers to adherence in adults with hemophilia in Poland.

Continuous Infusion of Factor VIII and von Willebrand Factor in Surgery: Trials with pdFVIII LFB or pdVWF LFB in Patients with Bleeding Disorders.

BACKGROUND: A plasma-derived factor VIII product (pdFVIII; Factane 100 or 200 IU/mL) and a plasma-derived von Willebrand factor product (pdVWF; Wilfactin 100 IU/mL) are approved for replacement therapy by intravenous bolus injections in hemophilia A (HA) and von Willebrand disease (VWD), respectively. However, in situations requiring intensive treatment, continuous infusion (CI) may be desirable to better control target plasma factor levels. AIM: To evaluate the perioperative hemostatic efficacy and safety of these concentrates administered by CI. METHODS: Three phase III trials were conducted. Adults with HA (FVIII:C < 1%) (studies 1 and 2) or VWD (VWF:RCo < 20%) (Study 3) received a preoperative bolus followed by CI of undiluted concentrate for at least 6 days. Bolus doses and CI rates were based on individual recovery and clearance, respectively. The initial infusion rate had to be higher for 48 hours for HA and 24 hours for VWD patients to anticipate potential fluctuations of factor concentrations during major surgery. Target levels of FVIII:C in HA and VWF:RCo in VWD were 80 and 70 IU/dL, respectively. Efficacy was assessed using a global hemostatic efficacy score. RESULTS: Studies 1, 2, and 3 included 12, 4, and 6 patients, respectively. Efficacy outcomes were excellent/good in all 22 major surgeries including 18 orthopedic procedures. Most daily measured FVIII and VWF levels (92%) were on target. No safety concerns, thrombotic events, or inhibitors were identified. CONCLUSION: pdFVIII and pdVWF administered by CI represent an effective and safe alternative to bolus injections in patients with severe HA or VWD undergoing surgery.

Orthopedic procedures in patients with congenital coagulation disorders: single center experience.

BACKGROUND: Spontaneous intraarticular bleeds in congenital coagulation disorders result in early and extensive damage to the joints and periarticular structures. Total arthroplasty is the only effective method of treating these defects. Interim surgical procedures (arthroscopy, osteotomy, etc.) exist that can postpone arthroplasty, especially considering the fact that the condition affects young people. The aim of this paper is to discuss the range of trauma care and orthopedic procedures performed in patients with congenital coagulation disorders. Also presented are early results of joint arthroplasty in these patients. MATERIAL AND METHODS: A total of 168 trauma care and orthopedic procedures were performed in patients with congenital coagulation disorders at the Clinical Department of Orthopedics and Traumatology of the Central Clinical Hospital of the Ministry of Interior in Warsaw in the years 2010-2013. Among them were total arthroplasties (79 arthroplasties of the knee, 30 of the hip, 3 of the ankle and 1 of the elbow), arthroscopies, filling bone cysts with grafts and trauma procedures. The HHS, KSS, AOFAS and MEPS scales were used to evaluate the respective clinical results of hip, knee, ankle and elbow arthroplasty procedures. A VAS was used to evaluate pain intensity. In knee arthroplasty patients, quality of life parameters were evaluated with the WOMAC index. RESULTS: In patients post hip arthroplasty, HHS scores increased by 50.22 points and VAS scores increased by 6.34 points. An increase of 116.41 points in KSS scores and 6.67 points in VAS scores was recorded in patients after knee arthroplasty. Also, WOMAC scores improved by 53.8 points after surgery. Evaluation of early results of ankle arthroplasty in the AOFAS scale showed a mean improvement of 35.5 points and a 5-point improvement in VAS scores. MEPS scores, used for evaluation of elbow arthroplasty results, improved from 15 to 70 points, with an improvement from 6 to 2 points in VAS scores. CONCLUSIONS: 1. Orthopedic procedures in patients with congenital coagulation disorders require thorough preparation of the patient and close cooperation between the orthopedic and hematological teams. 2. Early clinical outcomes are promising. 3. Decreased pain intensity, increased joint range of motion and improved quality of life post-surgery are observed.