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We have recently described an unusual situation which involved a combination of a factor IX and a protein C deficiency in a young child who presented, according to the bleeding tendency, as a hemophilia B patient. In this particular hemophiliac, baseline prothrombin fragment F1 + 2 levels were unexpectedly elevated and increased after an injection of a very high purity factor IX concentrate. This observation raised a question regarding the substitution schedule in the case of repeated injections of factor IX, since the thrombotic tendency has been a major concern with some factor IX concentrates. We monitored factor IX, prothrombin fragment F1 + 2, and D-dimer plasma levels before and during the 6 hr following the injection of an immunopurified factor IX concentrate. The results showed an increase in the F1 + 2 levels after the factor IX injection, but an increase lower than previously observed with an ion-exchange chromatography-purified concentrate. Furthermore, the F1 + 2 level returned to baseline value 6 hr after administration. This factor IX concentrate seems to be best for use in the patient where repeated injections are involved (as employed during surgery). Moreover, the data point out the advantage of a monoclonal antibody-purified factor IX concentrate over less purified concentrates in a specific situation, with regard to the thrombogenic risk.

Abstract 3810 Introduction: Despite frequent anemia and multiple transfusions during AML chemotherapy and allogeneic hematopoietic stem cell transplantation (allo-HSCT), recommendations and marketing authorization for erythropoietin (EPO) use are still missing. In the current prospective study, as primary objective, we evaluated the effect of EPO on patient's quality of life (QOL). Secondary objective was hemoglobin (Hb) recovery. In addition, a paired matched analysis using similar population was conducted to compare platelets (Pt) and red blood cells (RBC) transfusion number. Materials and methods: We included adult patients with Hb level ≤11g/dl induced by 1, 2 or 3 consolidation chemotherapy for AML in complete remission (CR) (group 1); or by allo-HSCT for any hematological disease (group 2). EPO was administered Sc. once per week during a maximum period of 6 months: for group 1, ARANESP® 150μg; for group 2, NEORECORMON® 30000IU; Hb level was monitored every week. Injections were stopped once the Hb level reached 12g/dl without any transfusion. If after 4 injections, no improvement was observed, doses were doubled, and if after 8 injections, no improvement was observed, patient was taken off-study for EPO inefficiency. The QOL was measured at baseline, at 1, 2, 3 and 6 months by the Functional Assessment of Cancer Therapy–Anemia (FACT–An). EPO responders patients were defined as having Hb level ≥12g/dl (EPO CR) or a ≥ 2g/dl increase [EPO partial response (EPO PR)] compared with baseline value without any transfusion requirement. The matching analysis took into account: sex, age, disease status, for the two groups, associated to cytogenetics, type of chemotherapy, sequential chemotherapy number for group 1, and diagnosis, conditioning, HSC source, number of previous transplants and GVHD for group 2. Results: Between April 2006 and December 2009, among 261 screened patients, 55 were included in group1 and 61 in group 2, patient characteristics for each group are summarized in Table1. Main exclusion criteria were EPO contra-indication and patient refusal. The median number of EPO injections/patient was 13 (3 – 24) in group1 and 8 (2 - 28) in group 2. For the global population (111 evaluable patients [52 group1 and 59 in group 2]), we have noticed a significant improvement of QOL during the 6 months follow-up according to FACT-An anemia (p=0.01). Despite a non-significant improvement for FACT-G, we observed a significant improvement in physical well-being (p Conclusion: This prospective study showed a real benefit of EPO administration on QOL, an achievement of a normal Hb level and a significant spare of RBC and Pt transfusions. Young AML patients, male allo-HSCT recipient, ABO compatible pairs seem to be the best candidates to benefit from EPO administration, with low AEs and no impact on OS or EFS. A cost-effectiveness study is ongoing and results will be communicated. Disclosures: No relevant conflicts of interest to declare.

This analysis studied 197 autologous HSCT performed in 132 patients treated for multiple myeloma (MM) in our center between 2000 and 2007. There were 53 females and 79 males with a median age of 56.8 years (34–72). At diagnosis there were 71 IgG (49κ, 22λ), 26 IgA (15κ,11λ), 2 IgD (1κ, 1λ), 27 light chain (18κ,9λ), 2 plasma cell leukemia, 3 non secretory, 1 non secretory/non excretory. There were 11 stage I (10A and 1B), 12 IIA, 96 III(75A and 21B) and 13 not classified. At diagnosis, 24/98pts had a del(13), and 65/179 had high levels of β2microglobulin. Before 2004, 50% of transplants were done and the median interval between diagnosis and HSCT was 7.8 months (3.5–131). We divided the population into 2 groups (I = 65: double-auto, II = 67 simple auto). The disease status pre-transplant according to the number of apheresis were: 1 apheresis (group I: 3CR, 29PR, 3SD, 1PD; group II: 2CR, 24PR, 3PD), 2 apheresis (group I: 16PR, 1SD, 1PD; group II: 3CR, 9PR, 1PD), 3 apheresis (group I: 1CR, 4PR; group II: 10PR, 1unknown), 4 apheresis (group I: 4PR, 1PD; group II: 1CR, 10PR, 2SD), 6 apheresis (group II: 1PR) and 8 apheresis (group: 1PR). PBSC were mobilized in steady state in 135 cases, 53 after cyclophosphamide alone, 9 cyclophosphamide with other drugs. During mobilization, we used GCSF in 179cases, GM-CSF in 5cases, SCF in 4 cases and associations of GM-CSF+ GCSF in 2(1%) cases and SCF+GCSF in 7(3.5%) cases. The median number of infused cells were: TNC 5×107/kg (1–59), CFU-GM 70.5×104/kg (0–2616) and CD34+cells 3×106/kg (0–27). Of these, 115 (58%) had a number of CD34+cells a conditional logistic-regression model to analyze associations between these variables and length of hospitalization, number of RBC and Pt transfusions; a multivariate analysis using Cox model to analyze the impact of these variables on length of aplasia ( We observed no significant impact of all studied variables on length of hospitalization and RBC transfusions and a significant negative impact of long interval diagnosis-T (p=0.05) and of the period > 2004 on Pt transfusion number (p=0.03). We showed a significant positive impact of CFU-GM number [HR=1 (1000–1.002) (p=0.03)] and growth factor use after transplantation [HR=0.55 (0.36–0.85) (p=0.005)] on days