bluuebunny/crossref_metadata_2025_split huggingface dataset:
PS1053 IMPACT OF CYTOMEGALOVIRUS REACTIVATION ON RELAPSE RATE AFTER ALLOGENEIC STEM CELL TRANSPLANTATION IN PATIENTS WITH ACUTE MYELOBLASTIC LEUKEMIA
Authors: Abdeljelil N. Ben , Hmida M. Ben , Ouerghi R. , Lakhal A. , Fatmi R. El , Torjemane L. , Belloumi D. , Ladeb S. , Achour W. , Othman T. Ben 
Published: June 2019

Background:Cytomegalovirus (CMV) infection is a major complication after allogeneic stem cell transplantation (ASCT).Aims:The aim of the study was to evaluate the impact of CMV reactivation on the relapse rate after ASCT in patients with acute myeloid leukemia (AML).Methods:Retrospective study conducted in patients with AML who underwent ASCT from HLA identical sibling donor between January 2011 and December 2018. Conditioning regimen consisted of Busulfex and Cyclophosphamide (Bu/Cy) or Fludarabine and Busulfex (F/Bu). Graft‐versus‐host disease (GVHD) prophylaxis consisted of cyclosporine and a short course of methotrexate. Antiviral prophylaxis for CMV infection was assured by Acyclovir from day +1 to day +180. CMV detection was carried out once‐a‐week from engraftment to day +100 by either pp 65 antigenemia test or real‐time quantitative PCR.Results:Ninety‐three patients were enrolled (55 men and 38 women). Median age was 33 years (range, 5 ‐ 49 y). At the time of transplant, 73 patients (78.5%) were in CR1, 16 patients (17.2%) were in CR2 and 4 patients (4.3%) were in response failure. CMV serostatus for donor (D) and recipient (R) was available for 44 cases (47.3%). Serostatus of R+/D+ and R+/D− was observed in 38 patients (86.4%). Stem cell source were BM in 49 cases (52.7%) and PBSC in 44 cases (47.3%). No graft failure was observed. Acute GVHD grade II‐IV occurred in 19 patients (20.4%). Chronic GVHD was observed in 40 patients (45.4%). Twenty‐nine patients (31.2%) developed CMV reactivation. With a median follow‐up of 2 years (range 49 days – 7 years), the overall survival (OS) and the non‐relapse mortality (NRM) were not statistically significant between patients with CMV reactivation and those without CMV reactivation (74% vs 63%, p = 0.3 and 20.7% vs 7.8%, p = 0.08, respectively). Twenty‐four patients (25.8%) relapsed at a median of 6 months (range 2 ‐ 67 months). the rate of relapse among patients with CMV reactivation was significantly lower than in those without CMV reactivation (10.3% vs 32.8%, p = 0.02). In univariate analysis, the CMV reactivation was the only factor associated with a decreased risk of relapse (OR = 0.23, 95% CI: 0.06–0.87, p = 0.02).Summary/Conclusion:CMV reactivation was associated with decreased relapse risk after ASCT for patients with AML without a benefit in OS.
PS1056 ACUTE MYELOID LEUKEMIA OR MYELODYSPLASTIC SYNDROME WITH CHROMOSOME 17 ABNORMALITIES AND LONG‐TERM OUTCOMES WITH OR WITHOUT HEMATOPOIETIC STEM CELL TRANSPLANTATION
Authors: Mohyuddin G.R. , Britt A. , Skikne B. , Singh A. , Lin T. , McGuirk J. , McClune B. , Ganguly S. , Abhyankar S. , Shune L. , Dunavin N. , Dias A. 
Published: June 2019

Background:Chromosome 17 (ch17) abnormalities, especially loss of the 17p region and P53 gene mutations (p53mut) result in very low rates of cure for patients with acute myeloid leukemia (AML) treated with conventional chemotherapy or allogenic stem cell transplant (ASCT).Aims:We assessed whether such poor outcomes extend to patients with all types of ch17 abnormalities and whether p53mut has further prognostic value.Methods:We retrospectively analyzed 98 patients at our institution with AML/myelodysplastic syndrome (MDS) who had ch17 abnormalities noted on cytogenetics. p53mut was analyzed by next generation sequencing (NGS).Results:61 patients with AML and 37 patients with MDS were included. Patient characteristics are in Table 1. 95 out of the 98 patients had 17p abnormalities as part of a complex karyotype, whereas 67 out of the 98 patients had a monosomal karyotype.Complete remission (CR) with first line treatment was similar between induction chemotherapy or hypomethylating agents, 22.9% vs 21.6 % (p = 0.33).Median overall survival (OS) for all patients was 10 months. Ch17 abnormalities were classified into those resulting in deletion of the p53 loci such as 17p deletion and monosomy 17 (17pdel/mono17) and all other ch17 abnormalities (ch17oth). Amongst patients with 17pdel/mono17with NGS available, 19 of 26 had p53mut, while for ch17oth, 13 of 17 had p53mut (p = 0.72).Patients having both p53mut and abnormal ch17 exhibited poorer OS compared to patients with no p53mut (10 vs 23 months, p = 0.02).30 patients (19 AML, 11 MDS) underwent ASCT, with a median post‐transplant OS of 11 months. For AML ASCT patients, 18 were in CR (13 with cytogenetic remission) and 1 had refractory disease at transplant, whereas for MDS patients, 3 were in CR (2 with cytogenetic remission) and 8 had stable disease. The post‐ASCT survival between AML/MDS cohorts did not differ (p = 0.6), although cytogenetic CR at time of ASCT trended towards improved OS (17 vs 8 months; p = 0.6). At last follow‐up, only 5 of 30 patients were alive, 4 in CR.Summary/Conclusion:AML/MDS patients with all types of ch17 abnormalities have a poor prognosis with or without ASCT, representing a distinct subset of patients amongst that that have complex cytogenetics. TP53 abnormalities detected by NGS are not limited to patients with 17pdel/mono17 and can be seen with other ch17 abnormalities. Drugs targeting the TP53 pathway and improving depth of response prior to ASCT are needed for improved outcomes in these patients.image
PS1059 DEFINING TRANSIENT ABNORMAL MYELOPOIESIS BY GATA1 MUTATION IN INFANTS WITH DOWN SYNDROME
Authors: Elalfy M. , Ragab I. , Abdel‐Khalek H. , Fayek M. , Kamal T. , Abdel‐Fattah N. 
Published: June 2019

Background:Infants with Down syndrome are more prone to transient abnormal myelopoiesis. A small percentage of this condition can be fatal, yet they are more prone to develop acute myeloid leukemia if harboring GATA1 mutation.Aims:Aim of the Work was to screen infants with Down syndrome for peripheral GATA1 mutation by sequencing of exon2 and to correlate it with clinical and laboratory parameters.Methods:The study included 79 patients with Down syndrome aged 1–90 days from the outpatient Genetics Clinic, Children's Hospital, Ain Shams University. Complete blood count with manual differential leukocytes count and blast cells count was assessed to all patients.By peripheral blast count patients were divided into three groups, Group A with peripheral blast count more than 10%, group B with blast count >0–10% and group C with no detected blasts in peripheral blood. Mutation analysis of exon2 of GATA1 gene was performed by Sanger sequencing to 15 cases based on results of blast cells count.Results:The median age of patients was 23.5 days. Prematurity was detected in only 6.3% of patients. Eighteen percent of DS patients (15 of 78) had blasts on blood smears (range 1%>60%). %). Group A included 4 patients, group B 11 patients and Group C 63 patients. The patients in group A were younger in age than patients in group B and C. Those 15 patients were divided into two groups: Mutation positive group: 4 patients, all of them had blast cells count >10% and Mutation negative group: 11 patients. The peripheral blast cells% was significantly higher in the mutation positive than mutation negative group. The only patient presented with splenomegaly was positive for the mutation of exon 2 of GATA‐1 gene.There was higher leucocytic count in patients with positive mutations compared to negative ones.One patient with positive GATA1 mutation developed AMKL and died of disease, he had initial blast count more than 10% in peripheral blood.Summary/Conclusion:All patients with positive GATA1 mutations had blasts>10% in their peripheral blood. One of the mutation positive patients developed acute myeloid leukemia. We conclude that blast cells count in peripheral blood combined with peripheral GATA1 mutation analysis can be used for close follow up of patients for early detection of acute megakaryocytic leukemia in infants with Down syndrome.
PS1080 LYMPHOMA ASSOCIATED HEMOPHAGOCYTIC SYNDROME: A SINGLE‐CENTER EXPERIENCE
Authors: Rojas Martínez J.A. , Prats‐Martín C. , Pérez Ortega L. , Morales Camacho R. , Knight Asorey T. , Pérez‐López O. , Caballero‐Velázquez T. , De la Cruz‐Vicente F. , Vargas de los Monteros M.T. , Bernal Ruiz R. , Pérez‐Simón J.A. 
Published: June 2019

Background:Aproximately 1% of the patients with haematological malignancies will develope an hemophagocytic syndrome. Some types of lymphoma are associated with higher risks of developing hemophagocytic syndrome. It is described a high rate of lymphoma associated hemophagocytic syndrome (LAHS) related‐mortality. Only few studies have addressed LAHS.Aims:To describe the clinical and epidemiological features and the prognosis of LAHS in a tertiary hospital from January 2008 to December 2018.Methods:We performed a ambispective observational study of the cases of hemophagocytic lymphohistiocytosis (HLH) diagnosed by bone marrow aspirate. The diagnosis was made according to the diagnostic criteria of the International Histiocyte Society (IHS‐2004). Lymphoma classification followed the World Health Organization (WHO) 2017 system. Epidemiological and clinical features of the patients diagnosed of LAHS were analysed. Statistics performed with SPSS 17.0.imageResults:48 patients were diagnosed with HLH in our center in this period. 16 cases (37%) were associated to lymphoma. The median age for patients diagnosed with LAHS was 63 (range: 13–80). The most common histological type was diffuse large B cell lymphoma, which occurred in 7 of 16 patients (44 %). Peripheral T cell lymphoma and Hodgkin lymphoma occurred both in 3 of 16 patients. 7 patients (44%) had other known risk factor for LAHS: viral infection, autoimmune disease and immunosuppression. The clinical and analytical features are summarized in Table 1. The histopathological analysis of the bone marrow did not identify hemophagocytosis in any cases.Most of the patients diagnosed with LAHS (9 of 14) were treated with the first‐line chemotherapy regimen for lymphoma. 3 patients were treated following the HLH‐2004 protocol. 2 patients died before receiving any treatment. The median overall survival time was 88 days (range: 0 days‐6 years). The three months mortality rate was 50%. 4 patients are still alive with 2, 3, 35 and 71 months after the diagnosis of LAHS (Fig 1).imageSummary/Conclusion:The most common associated lymphoma was diffuse large B cell lymphoma. 44% of the patients had other known risk factor for LAHS. The bone marrow aspirate is superior to the histopathological examination for the diagnosis of LAHS. The three months mortality rate was 50%. Further researches are needed to gain better understanding about this entity.
PS1085 ORAL CHOP LIKE CHEMOTHERAPY IN ELDERLY PATIENTS WITH HIGH‐GRADE NON HODGKIN B CELL LYMPHOMA
Authors: Guidez S. , Tomowiak C. , Princet I. , Motard C. , Corby A. , Machet A. , Debiais C. , Guilhot J. , Delwail V. 
Published: June 2019

Background:The Doxorubicin based R‐CHOP regimen remains the standard of care for the upfront treatment of DLBCL. The advent of oral idarubicin permitted to develop an all‐oral ambulatory oro‐CIEP regimen.Aims:We hypothesized this regimen would improve the treatment of elderly DLBCL.Methods:We have performed a phase 1/2 study to determine the MTD[CA1] of idarubicin in the oro‐CIEP regimen. Idarubicin was orally administrated on day 1 with starting dose at 20 mg/m2 and escalated per 10 mg/m2 increment.Results:Twenty‐six newly diagnosed patients were enrolled, median age of 71 years (range, 62–80). All first 3 patients developed a DLT at level 3 (40 mg/m2), and 14 patients were then enrolled at RP2D 30 mg/m2 in the phase 2. The most significant hematological toxicities were thrombocytopenia grade 3 or 4 (3.3%) and neutropenia grade 3 or 4 (26%); and extra‐hematological toxicities were infectious disease (5% grade 3 or 4). Second primary malignancies were reported in three patients. No toxic related death was observed. ORR and CR rates were 85% and 77%, respectively. The 5‐year overall survival and event free survival were 65% (95%CI 41;81) and 65% (95%CI 42;81), respectively.Summary/Conclusion:This analysis shows that oral idarubicin at the MTD of 30 mg/m2 is safe and active in elderly DLBCL. The results we have observed, particularly overall survival seemed to be superimposable to previous publications with the regular CHOP‐like regimens for this population. Further improvements in the protocol will be introduced with the subcutaneous anti‐CD20 immunotherapy approach. This data needs to be confirmed in the context of a larger study.
PS1104 EPIDEMIOLOGICAL STUDY OF IDIOPATHIC THROMBOCYTOPENIC PURPURA AMONG THE ADULT POPULATION OF THE RUSSIAN FEDERATION
Authors: Melikyan A. , Egorova E. , Pustovaya E. , Kolosheinova T. , Ladygina E. , Volodicheva E. , Kaporskaia T. , Ilyasov R. , Shelekhova T. , Fedorova N. , Zotova I. , Sycheva T. , Kontievskii I. , Shestopalova I. , Kurkina N. , Syrtseva E. , Tarasenko E. , Meresiy O. , Cherkashenko Z. , Chabaeva Y. , Kulikov S. 
Published: June 2019

Background:Primary immune thrombocytopenia (idiopathic thrombocytopenic purpura ‐ ITP) is a rare autoimmune disease, the main clinical manifestations of which are hemorrhagic syndrome of varying severity. The incidence of ITP in the adult population of different countries varies from 1.6 to 12.5/100,000 person‐years. ITP register started in the Russian Federation (RF) In December 2014. This is multicenter, prospective observational cohort study “Epidemiological and clinical characteristics of ITP in adults in Russia”.Aims:Assessment of the epidemiological characteristics of ITP in the adult population of the RF.Methods:Clinical and laboratory data of the register “Epidemiological and clinical characteristics of ITP in adults in Russia”. Methods of descriptive statistics, frequency, variance analysis and event analysis used in statistical processing.Results:The incidence of ITP in the adult population of the Russian Federation averaged 2.09/100,000 people. The highest morbidity of 2.7/100,000 recorded among women under 40 years of age. While in men there was a gradual increase in the incidence from 1.09 to 2.5/100,000 people over the age of 60 years. The result obtained is comparable with the data of the registers of other European countries. An analysis of incidence in the sex‐age strata showed that men have a tendency to an increase in the incidence of ITP from 1.09 at the age of 40, to 2.98 at the age of 60. Similar changes in the incidence in women of different age groups not detected. It is noteworthy that in the age group over 60 years, the incidence in men and women is almost the same, whereas data from epidemiological studies in other European countries showed a significant predominance in this group of men.There was a significant relationship between the risk of development, the degree of bleeding and the number of platelets in the onset of the disease: the severity of hemorrhagic syndrome increases with the number of platelets below 50x109/l (p < 0.0001). In 92.2% of cases glucocorticosteroid hormones prescribed as the first line of therapy. Analysis of methods of treatment used as a second line therapy demonstrated that the probability of splenectomy the last 3 years decreased from 26 to 17%, while the number of patients receiving thrombopoetin receptors agonists increased from 5.9 to 45.7%.Summary/Conclusion:The epidemiological data are comparable with those in other European countries. Geographical variations of incidence in different regions of the Russian Federation require further study.
PS1105 CONGENITAL DEFICIENCY OF COAGULATION FACTOR VII IN THE SOUTH OF TUNISIA: ABOUT 88 CASES
Authors: Kacem S. , Megdiche F. , Ghorbel M. , Maalej S. , Fakhfakh Y. , Kassar O. , Frikha I. , Kotti F. , Chaabane H. , Elloumi M. , Kallel C. 
Published: June 2019

Background:Congenital factor VII deficiency (FVII) is the least rare among uncommon hereditary bleeding disorders (1:500 000). This coagulopathy, inherited in an autosomal recessive pattern, is characterized by a bleeding tendency, varied prognosis and a lack of correlation between clinical phenotypes and biological analysis.Aims:The objective of our study was to describe and to explore the clinical and the biological features of FVII deficiency in the South of Tunisia.Methods:Our FVII‐deficient cohort consisted of 88 patients whose FVII activity was less than 50% and who were referred to our laboratory from January 2000 to December 2018 because of a prolonged prothrombin time (PT) or hemorrhagic manifestations. The FVII deficiency was considered as severe for FVII coagulant activities (FVII:C) <2%, moderate for rates between 2 and 19% and minor for values between 20 and 49%. The statistical analysis was based on Chi‐squared test.Results:The mean age of the patients was 22 years old ranging between 21 months and 63 years old and the sex ratio was about 0.83. Among 88 patients, belonging to 58 unrelated Tunisian families, 45 cases (51.2%) were diagnosed accidentally during presurgical screening, 28 cases (31.8%) were noticed following family screening and 15 patients (17%) were discovered due to bleeding accidents. Clinically, 64 patients (72.7%) were asymptomatic whereas the bleeding frequency was about 27.3%. Clinical examination revealed several bleeding manifestations as follows : gingivorrhagia noticed in 12 patients (50%), followed by epistaxis in nine patients (37.5%). Four cases had hematemesis (16.6%), hematoma was found in three (12.5%) and one of the patients had a cerebral haemorrhage (4.2%). Added to that, some bleeding signs, not commonly related to the FVII deficiency, had been noticed such as: menorrhagia, haemoperitoneum, hematuria and bruising. The laboratory severity of each patient was also determined : FVII deficiency was classified as severe in 3.4%, moderate in 14.8% and minor in 81.8% of cases. With reference to statistics, results showed a weak correlation between clinical data and biological levels of FVII:C (p = 0.004). In fact, 15 patients with a minor FVII deficiency had presented haemorrhages with a variable bleeding severity; while one of the patients was asymptomatic despite the severity of his defect.Summary/Conclusion:Our study, in accordance with the literature, revealed a great interindividual heterogeneity of the clinical phenotype of FVII deficiency. In addition, bleeding severity did not necessarily correlate with FVII:C and varies markedly between individuals with a similar FVII:C. This would explain the delay and the underestimation of the diagnosis. In addition, the family screening had a prominent effect in improving the earliness of the diagnosis and in ensuring an adequate monitoring of these Tunisian patients.
PS1106 A RETROSPECTIVE ANALYSIS OF IMMUNE THROMBOCYTOPENIA PATIENTS TREATMENT RESULTS AFTER SEQUENTIAL SWITCHING OF THROMBOPOIETIC RECEPTOR‐AGONISTS
Authors: Ptushkin V. , Vinogradova O. , Tkachenko N. , Pankrashkina M. , Chernikov M. , Perova V. , Shikhbabaeva D. 
Published: June 2019

Background:Immune thrombocytopenia (ITP) is an immune‐mediated disease characterized by increased platelet destruction and impaired platelet production with transient or persistent decrease in the platelet count and increased risk of bleeding. Romiplostim (R) and Eltrombopag (E) are the first thrombopoietic receptor‐agonists (TPO‐ra) with demonstrated efficacy against ITP. So far, very few data are available about the potential contribution of switching from romiplostim to eltrombopag or vice versa in terms of efficacy or tolerance.Aims:To evaluate the reasons for and result of TPO‐ra switching in adult ITP patientsMethods:We retrospectively analyzed all patients who received sequential treatment with both TPO‐RA between 2014 and 2018 in Moscow city hematology center (Botkin hospital).Results:Thirty‐three pts with primary ITP were included; 19 received first Romiplostim and 15 received first Eltrombopag. Reasons for switching were inefficacy (n = 30) and adverse events (n = 3). All patients (33) had previously received multiple courses of corticosteroids and IV Ig infusions, four (10%) received immunosuppression (rituximab), and five (14%) performed splenectomy. The median platelet count at the administration of the second TPO‐ra was 17x109/l (range 3–25x109/l). Twenty‐five (76%) patients had bleeding symptoms before the second TPO‐ra administration.From 33 included patients partial response (> 50x109/l without platelet transfusions and bleeding) was achieved in 16 (70%). PR was seen in sixteen from 19 (84%) in E to R group and seven from 15 (47%) in R to E group. Complete response (>100x109/l without pl. transfusions and bleeding) were achieve in 17 from 33 (52%). Twelve pts from 19 (63%) achieve CR in E to R group and five from 15 (30%) in R to E group. Two of 17 (10%) pts achieved a CR within 1–3 months, 11 (67%) pts in 3–12 months, and four (23%) pts later than 1 year from the start of switching to second TPO‐ra therapy.Three pts (2 in E group due to repeated thrombosis, and 1 in R group due to persistent increased liver enzymes) with recommendation of switching to second TPO‐ra due to adverse events resolved rapidly and maintained remission on alternative drug.In nine pts (28%) the switching to second TPO‐ra therapy was ineffective (blood platelet level was less than 30x109/l pl). All of them were resistant to first TPO‐ra.Summary/Conclusion:Our results confirmed that switching from one thrombopoietic receptor‐agonist to another could be beneficial for patients with chronic immune thrombopenia who failed to respond. Yet longer follow‐up and larger number of pts enrolled are required to fully assess the potential benefits of such therapy change.
PS1180 FOLLOW‐UP OF LOWER DOSE DASATINIB 50MG DAILY AS FRONTLINE THERAPY IN NEWLY DIAGNOSED CHRONIC PHASE CHRONIC MYELOID LEUKEMIA (CML‐CP)
Authors: Naqvi K. , Jabbour E. , Skinner J. , Anderson K. , Yilmaz M. , Ferrajoli A. , Bose P. , Thompson P. , Alvarado Y. , Jain N. , Takahashi K. , Burger J. , Estrov Z. , Borthakur G. , Pemmaraju N. , Dellasala S. , Paul S. , Cortes J. , Kantarjian H. 
Published: June 2019

Background:Dasatinib, a potent BCR‐ABL tyrosine kinase inhibitor (TKI), is approved for the treatment of chronic phase CML (CML‐CP) in the frontline and salvage settings. Notable side effects include pleural effusions and myelosuppression. We previously reported dasatinib 50 mg daily to be active and better tolerated than the approved 100 mg daily dose (CANCER. 2018 Jul 1;124(13):2740–2747).Aims:An update on the efficacy and toxicity profile of lower dose dasatinib 50 mg orally daily in patients with early CML‐CP.Methods:All patients presenting to our institution in early CML‐CP were eligible to participate. Prior TKI therapy for up to 1 month was allowed. Responses were assessed according to the European LeukemiaNet guidelines (Baccarani et al. Blood 2013 122.872:884).Results:From March 2016 to March 2018, 81 patients have been enrolled. Median age is 47 years (20–84). Patients categorized by Sokal risk are: low 53; intermediate 22 and high 6. Median follow‐up is 18 months (9–31). Cumulative response rates (n = 81) over time are shown in the gragh below.At 3 months, 96% patients achieved early molecular response (BCR‐ABL PCR ≤10%). Median time to CCyR was 4.6 months, MMR 6.0 months, MR4.0 11.4 months and MR4.5 12.2 months. Eighteen patients had treatment interruption: pleural effusion 4 (possibly related 3, unrelated 1 due to pneumonia); gastrointestinal bleed 2; thrombocytopenia 3; transaminitis 2; renal dysfunction 1; asthma exacerbation 1; pneumonitis 1; lower extremity edema 1; myalgias 1, and pregnancy 2. Four patients had dose reductions: pleural effusion 3; myalgias 1. Four patients had dasatinib dose increased to 100 mg: lack of CCyR at 6 months, 3; lack of MMR at 12 months, 1. Four patients are off study: no response 2, pneumonitis 1, and insurance 1. None of the patients have transformed or died.Summary/Conclusion:These updated results continue to support dasatinib 50 mg daily as an effective and safe dose for early CML‐CP.image