: Factor XIII deficiency is a rare autosomal recessive disorder of hemostasis characterized by a plasmatic factor XIII level less than 1% in homozygote and bleeding as of the youth. The aim of the study is to describe the clinical features and the outcome of the patients and to determine molecular characteristics. A retrospective study, was conducted on seven patients with factor XIII deficiency in the department of hematology and pediatrics, Hedi Chaker Hospital, Sfax, Tunisia during the period of 14 years (2001-2014). The activity of factor XIII in plasma of the patients was less than 1%. Seven patients from five unrelated families were recorded (four men and three women). Median age at diagnosis was 3.5 years. All patients had consanguineous parents. Six patients presented umbilical bleeding and only three patients had intracranial bleeding. Other bleeding features were seen, including skin and mucosal bleeding, muscular hematoma, and splenic rupture. Recurrent abortions were observed in one patient. The standard screening tests were normal. Genetic analysis identified two mutations interesting the subunit A of factor XIII. All patients received transfusion of fresh frozen plasma monthly. One patient was died because of intracranial hemorrhage.Factor XIII deficiency is a rare bleeding disorder which frequently increases in areas with high consanguinity. In our study, we identified a founder mutation. The prognosis of the disorder is related to hemorrhagic complications especially to life-threatening intracranial bleeding. Prophylaxis consists of factor XIII concentrate or recombinant factor XIII. If these are unavailable, fresh frozen plasma may be used.
Background: High dose Melphalan (HDM) and autologous stem cell transplantation (ASCT) is a standard care for Myeloma ≤ 65 years. Studies have demonstrated that depth of response prior to ASCT does not impact outcome post ASCT, e.g. solely depth of response post ASCT matters. However, with improving induction regimens, the questions remains whether patients achieving a deeper response at completion of induction would not perform better post ASCT. ASCT can only be performed in Tunisia if newly diagnosed Multiple Myeloma (NDMM) achieves at least PR; we therefore sought to report the national Tunisian experience. Patients and Methods: NDMM aged ≤65 years received three cycles of Thalidomide (200mg daily) and Dexamethasone, followed by HDM and ASCT.52% received maintenance therapy for 12 months with Thalidomide, from 3 months post transplantation. Non responders to TD induction were salvaged with Lenalidomide or Bortezomib-based regimen. The response was assessed based on IMWG response criteria. The study is performed in ITT. Results: 202 consecutive pts were included between April 2012 and December 2014. The median age was 56 yrs (range, 25-65), sex ratio was 1.R-ISS stage was 3 in 27.5%. 21% had high risk cytogenetic (by Conventional karyotyping and FISH). Renal failure was observed in 17%. ORR after induction was 71% with 12% CR, 17% VGPR. 22% failed to obtain PR following TD induction, and 51% of whom received salvage induction therapy (ST) with 72% that further reached ≥PR after ST. Overall, 141 pts (70%) underwent ASCT, 121 of whom had evidence of chemo-sensitive MM at completion of induction. 16% were transplanted in CR, 22% in VGPR and 62% in PR. At 3 months post-ASCT, the ORR in induction chemo-sensitive MM was 89%: 36% CR, 20% VGPR, and 33% PR. With a median follow-up post-ASCT of 27 months, the OS, PFS and EFS at 27 months were 82%, 60.5% and 56%, respectively. Maintenance treatment was significantly associated with longer PFS only in MM who did not achieve CR (29 versus 9 months, p=0.004). MM with improved response post-ASCT had a significantly longer PFS (39 versus 21 months, p=0.003) and EFS (39 versus 20 months, p=0.002). Importantly, achieving CR before (p=0.03) and after ASCT (p<0.0001) was also predictive for prolonged EFS. Early relapses/progressions (less than 18 months) was the sole predictive factor of adverse OS in our study (p=0.01). In multivariate analysis, ISS stage 3 was the sole independent predictor of induction failure (p=0.003). Importantly, predictive factors of achievement of CR post-ASCT comprised absence of delay farther to 4 months from completion of induction (p=0.006; OR = 4.54) and achievement of at least VGPR status before transplant (p=0,001; OR = 4.09). Conclusion: Achievement of at least VGPR at completion of induction improved response after ASCT and consequently influenced the post-ASCT outcome. Therefore, depth of response matters before and after ASCT, and validates in some extent the concept of induction salvage therapy prior to ASCT for patient not reaching response. Disclosures Leleu: TEVA: Membership on an entity's Board of Directors or advisory committees; Novartis: Honoraria; LeoPharma: Honoraria; Pierre Fabre: Honoraria; Amgen: Honoraria; Bristol-Myers Squibb: Honoraria; Takeda: Honoraria; Celgene: Honoraria; Janssen: Honoraria.
Background: Measurement of minimal residual disease (MRD) in B-cell acute lymphoblastic leukemia (B-ALL) is integrated in treatment protocols due to it prognostic significance. In Ph+ B-ALL BCR-ABL1 transcripts are the preferential molecular targets for MRD assessment.
Background:Acute lymphoblastic leukemia (ALL) had a good prognostic in children and young adults. However, some parameters were predictive of a worse outcome, particularly the poor response to prephase.Aims:We analysed in this study the impact of persistent blasts at day 8 of pre‐phase in cortico‐sensitives patients with ALL.Methods:Between january 2000 and December 2015, we retrospectively analysed the data of 264 children and young adults treated according the EORTC 58951 protocol. Cortico‐sensitivity (Cs) was considered if peripheral blast count is less than 1000/mm3 at day 8 of pre‐phase. The group of cortico‐sensitives ALL was subdivided on two subgroups : the first with no blasts at day 8 (Cs = 0) and the second with more (Csǂ0). We analysed the impact of cortico‐sensitivity on complete remission (CR), relapse and survival (overall survival (OS), event free survival (EFS) and disease free survival (DFS)).Results:One hundred ninety two children and 72 young adults were treated according to the EORTC 58951 protocol. Median age was 10 years (1 to 30 years). Sex ratio was 1,5. B and T phenotype were observed in respectively 64 and 36% of cases. The ALL were stratified in low risk (11 patients), average risk (182 patients), and high risk (71 patients). Two hundred and nine patients (79%) were cortico‐sensitive, among them 150 patients (72%) had no blast at day 8 of pre‐phase. The therapeutics results in our study and into the two groups of cortico‐sensitivity were shown in this table :Summary/Conclusion:Cortico‐sensitivity is one of several parameters that predict good outcome in ALL. Moreover, our study showed that no blasts in peripheral blood smears at day 8of prephase can be considered a good first marker of early blastic clearance and good outcome. In fact, this parameter does not discussed previously in the literature.image
Qi and blood stasis, phlegm and blood stasis are intertwined.According to the theory of "Wei Qi Ying Xue" from the "Fu Qi Qi Ying Xue" to treat and detoxify.Metronomic chemotherapy has good tolerance and prolongation of survival in the treatment of malignant tumor. Modern pharmacological studies have found that many Chinese herbs for clearing away heat and detoxification could inhibit the proliferation of lymphoma cells, inhibit angiogenesis and regulate immune function. Fully accord with the concept of metronomic chemotherapy, the combination of Qingrejiedu decoction and oral chemotherapy is a new combination of metronomic chemotherapy. In order to verify the efficacy of lowdose continuous oral chemotherapy combined with Qingrejiedu decoction, we made a preliminary attempt for the treatment of elderly lymphoma with refractory or recurrence.Methods: Sixty-three cases of refractory or relapsed non-Hodgkin's lymphoma were treated with metronomic chemotherapy combined with Qingrejiedu decoction and Qingrejiedu decoction, and were treated with Qingrejiedu decoction and metronomic chemotherapy.Aged 66 to 93 years, median age 74 years, male 37 cases, female 26 cases. According to the Chinese medicine diagnosis, 3 cases of ABSTRACT 449 Background: There is still no clear therapeutic algorithm for MALT lymphoma patients beyond H. pylori eradication and while recent data
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