Abstract Number: PB0972
Meeting: ISTH 2020 Congress
Background: Patients with non-severe hemophilia A may perioperatively be treated with desmopressin or factor VIII (FVIII) concentrate. Desmopressin leads to higher FVIII plasma levels, however, interpatient variability in FVIII rise is high. If FVIII increase is sufficient, minor procedures can be performed with desmopressin monotreatment. If insufficient, FVIII concentrate is used. Desmopressin combined with FVIII concentrate is not common practice. Because desmopressin is less expensive than concentrate, combined use may be cost-effective.
Aims: Assess accuracy, safety and cost-effectiveness of perioperative pharmacokinetic (PK)-guided combination treatment (desmopressin and FVIII concentrate) and FVIII concentrate savings in non-severe hemophilia patients.
Methods: Hemophilia A patients responsive to desmopressin undergoing a procedure were included. Desmopressin was administered intravenously (0.3 µg/kg). Based on previously determined FVIII response on desmopressin in the patient, additional FVIII concentrate was calculated using PK-guidance for a maximum of three days. FVIII plasma levels were determined before and after combination treatment (peak and trough levels). Hypothetical FVIII concentrate loading dose was calculated. A predicted FVIII level by the desmopressin-FVIII-PK-model was considered accurate if difference between measured and predicted FVIII level was < 0.2 IU/ml.
Results: Patient characteristics and savings of 18 procedures are described in table 1. Median age was 49.5 years (interquartile range 39.8 – 59.3). Figure 1 describes PK-model accuracy for FVIII peak level on day 0 and trough levels on day 1 and 2. At day 0, 10/18 (55.5%) peak level predictions were accurate. At day 1, 11/15 (73.3%) trough level measurements were accurate and at day 2, 10/13 (76%). Modelling technique was continously adapted and improved based on study data, leading to better predictions.
Conclusions: Perioperative combination treatment of desmopressin and FVIII concentrate under PK guidance in non-severe hemophilia A patients provides an accurate dosing advice in the majority of patients. In addition, this resulted in considerable FVIII concentrate savings.
|Hemophilia severity |
|Lowest measured FVIII (IU/ml)||0.13$ (0.675 – 0.25)|
|Loading dose FVIII savings (IU)||1375 (875 – 2000)|
|Bleeding risk procedure |
|Performed procedure |
– Oral and maxillofacial
|Days of combination treatment |
|Bleeding complications||2 (11.1)|
|$ Reported in median and interquartile range (Q1 – Q3)|
* One patient had a discectomy L5-S1 and a molar extraction
To cite this abstract in AMA style:Romano LGR, Schütte LM, van Hest RM, Meijer K, Laros-van Gorkom BAP, Nieuwenhuizen L, Eikenboom J, Moenen FCJI, Uitslager N, Coppens M, Cnossen MH, Fijnvandraat K, Driessens MHE, Polinder S, Leebeek FWG, Mahtôt RAA, Kruip MJHA. “Best of Both Worlds”: Combination Treatment of Desmopressin and FVIII Concentrates in Hemophilia Patients Optimizes Therapy and Reduces Costs [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/best-of-both-worlds-combination-treatment-of-desmopressin-and-fviii-concentrates-in-hemophilia-patients-optimizes-therapy-and-reduces-costs/. Accessed January 18, 2021.
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