The clinicolaboratory characteristics and treatment outcome of 10 Korean patients with factor V deficiency

초록

Aims: We retrospectively analysed the clinicolaboratory features and treatment outcomes of FV deficiency in 10 Korean patients. Methods: Between January 1987 and December 2012, nine case reports published in Korean journal or presented at Korea Society on Thrombosis and Hemostasis (KSTH) and one medical record in our institution were reviewed. Results: A total of 8 consecutive adult patients, female (50%),median (range) age 50 (24-71) years, median (range) baseline platelet count 19 (3-32)x109/L.,median of 3 (2-4) prior ITP therapies,received eltrombopag with 50mg once daily starting dose to maintain platelet counts in the target range of 50-150x109/L.. The median time since ITP diagnosis was 3 years (range, 1-4 years) and none of the patients had undergone a splenectomy. The efficacy of oral eltrombopag was assessed after 6 weeks administration. All patients achieved a platelet count ≥50x109/L. and 6 out of 8 pts achieved CR (defined as platelet count ≥100 * 10^9/L measured on two occasions >7 days apart and the absence of bleeding). In 4 out of 8 patients occurred more dose adjustments and treatment interruptions to maintain platelet counts in the target range Moreover it was not possible, in this subset, to stop the treatment for a relapse within the first three weeks of discontinuation. For all these reasons a maintenance dose of 25 mg every 3 days starting on the ninth month. The median (range) follow-up of this case series was of 7 months (6-20), during which all patients maintained a safe platelet count without the need to increase the dose or frequency of administration. No adverse events were observed. Summary and Conclusions: Maintenance therapy was manageable and well tolerated.