Efficacy and safety of prophylactic treatment with plasma-derived factor XIII concentrate (human) in patients with congenital factor XIII deficiency
Ashley C, Chang E, Davis J, Mangione A, Frame V, Nugent DJ
UNLABELLED Congenital factor XIII (FXIII) deficiency is an extremely rare, potentially life-threatening bleeding disorder. Routine prophylactic management is recommended for individuals with clinically relevant FXIII deficiency. This prospective, multicentre, open-label study evaluated the long-term efficacy and safety of prophylactic infusions of FXIII concentrate (human) 40 IU kg(-1) in patients with congenital FXIII deficiency. FXIII concentrate (human) was administered every 4 weeks for 12 months. Dosing was adjusted to maintain trough FXIII activity levels of 5-20%. Logistical and ethical constraints precluded use of a placebo control group. Annualized incidence of spontaneous bleeding was compared with historical rates; safety was assessed as a secondary objective. Forty-one patients were enrolled and completed the study. The annualized rate for spontaneous bleeding episodes requiring FXIII treatment was 0.000 episodes per patient-year (95% CI: 0.000; 0.097). The study met its primary endpoint: the upper limit of the 95% CI was substantially below the historical rate of 2.5 bleeding episodes per patient-year. Five spontaneous bleeding episodes (involving three patients; none requiring FXIII treatment) and eight trauma-related bleeding episodes (two requiring FXIII treatment) occurred. Five patients had surgery during the study, only one of whom required FXIII treatment for post-surgical bleeding. Most patients (>85%) had trough FXIII activity levels >10%. No patient discontinued treatment due to an adverse event. No adverse events related to thromboembolism or viral transmission were reported. Prophylactic treatment with FXIII concentrate (human) was well tolerated and prevented spontaneous bleeding episodes that were serious enough to require treatment with FXIII-containing product. CLINICAL TRIAL REGISTRATION www.clinicaltrials.gov/ct2/show/NCT00885742. 2014 The Authors. Haemophilia Published by John Wiley & Sons Ltd.
Transfusing neonates based on platelet count vs. platelet mass: A randomized feasibility-pilot study
Zisk JL, Mackley A, Clearly G, Chang E, Christensen RD, Paul DA
Abstract The objective of this study was to obtain pilot data on which to judge the feasibility and sample size needed for a future comparative-effectiveness trial of platelet transfusions in the NICU. We conducted a limited-scope pilot trial in which neonates were randomized to receive platelet transfusions based on platelet mass vs. platelet count, using preset "transfusion-trigger" values. Analysis included parental consent rate, number of platelet transfusions given, bleeding episodes recorded, and mortality rate. Statistical analysis included ANOVA and Chi-square. A convenience sample of 30 were randomized; 15 per group. No differences were found between groups in gestational age, birth weight, race, gender or clinical diagnoses. The study consent rate was 52% (30/58). No differences were found in number of platelet transfusions received, bleeding episodes, or mortality. Lack of a trend in transfusion-reduction resulted in inability to estimate the number needed in a future comparative-effectiveness trial. Using platelet mass, rather than platelet count, for a NICU platelet transfusion trigger is feasible. However, any future comparative-effectiveness trial, testing the hypothesis that a platelet mass-based trigger reduces the transfusion rate will likely require a very large sample size.
Recovery from deep-plane rhytidectomy following unilateral wound treatment with autologous platelet gel: a pilot study
Powell DM, Chang E, Farrior EH
Archives of Facial Plastic Surgery. 2001;3((4):):245-50.
OBJECTIVE To determine the effects of treatment with autologous platelet-rich plasma mixed with thrombin and calcium chloride to form an autologous platelet gel (APG) on postoperative recovery from deep-plane rhytidectomy. STUDY DESIGN A prospective, randomized, controlled pilot study. SETTING An accredited ambulatory facial plastic surgery center. PATIENTS Healthy volunteer women (N = 8) undergoing rhytidectomy. INTERVENTION Unilateral autologous platelet-rich plasma wound treatment during standard deep-plane rhytidectomy. MAIN OUTCOME MEASURES Staged postoperative facial photographs were graded in a blinded fashion by 3 facial plastic surgeon reviewers for postoperative ecchymosis and edema. Each facial side treated with APG that demonstrated less edema or ecchymosis than the non-APG-treated side was designated a positive response; otherwise, the response was equal (no difference) or negative (untreated side had less edema or ecchymosis). RESULTS Twenty-one positive and 21 equal responses were observed compared with 8 negative ones. Of 20 unanimous observations, 15 were positive, only 3 equal, and 1 negative. CONCLUSIONS Treatment with APG may prevent or improve edema or ecchymosis after deep-plane rhytidectomy. This trend is more apparent for ecchymosis than for edema, and is chiefly demonstrable in the early phases of recovery. These observations are consistent with previous reports of cell tissue culture and wound response to concentrated platelet product.