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12 th Congress of the European Hematology Association 0761 PLATELET RESPONSE TO THE ORAL PLATELET GROWTH FACTOR ELTROMBOPAG IN A PATIENT WITH MYH9 SYNDROME (MAY-HEGGLIN ANOMALY): A CASE REPORT A. Baranwal, 1 A. Di Feo, 2 J.K. Hon, 3 J. Martignetti, 2 M.A. Arning 1 1 GlaxoSmithKline, COLLEGEVILLE; 2 Mount Sinai School of Medicine, NEW YORK; 3 Clearview Cancer Institute, HUNTSVILLE, USA Background. May-Hegglin anomaly and Sebastian, Fechtner and Epstein syndromes are rare autosomal dominant disorders that are characterized by thrombocytopenia and giant platelets resulting from mutations of the MYH9 gene. Patients often have an increased bleeding tendency and require platelet transfusions when faced with a haemostatic challenge. Alport-like features, including nephropathy, cataracts and/or hearing loss, can also be present. Aims. To present a case report of platelet response in a woman with MYH9 syndrome treated with eltrombopag. Methods. The patient was enrolled in a Phase III eltrombopag study of patients with chronic idiopathic thrombocytopenic purpura (ITP) and platelet counts 200,000/µL within 3 weeks after initiation of eltrombopag. Clinical evidence of bruising disappeared during the period when platelet count was >20,000/µL (day 8). After discontinuing eltrombopag platelet count slowly decreased. During follow-up, it was found that bruising returned when the platelet count was 27,000/µL. Peripheral smears before and following treatment showed the presence of numerous giant platelets. Family medical history revealed that eight paternal family members had a history of macrothrombocytopenia. Therefore, molecular characterization was performed to define the genetic basis of the familial disorder by directly sequencing the MYH9 gene in the patient. Two mutations were identified that predict amino acid changes at R702W (exon 30) and R1933X (exon 40). Although amino acid substitutions at position R702 and truncation at R1933 have been documented, this is the first description of a patient having both mutations and substitution of a tryptophan to replace the arginine at position 702. Conclusions. This case report provides scientific rationale for future studies of the oral platelet growth factor eltrombopag in individuals with this inherited form of thrombocytopenia. Figure 1. 284 | haematologica/the hematology journal | 2007; 92(s1) Quality of life, palliative care 0762 PATIENT INFORMATION SHEETS FOR CLINICAL HEAMATOLOGY TRIALS ARE TOO COMPLEX FOR MOST PATIENTS TO UNDERSTAND R. Soutar, 1 S. Horn2 1 2 Department of Haematology, GLASGOW; University of Glasgow, GLAS- GOW, United Kingdom Background. Informed consent is a critical part of clinical research as protection for both the patient and the researcher. For consent to be valid the patient must be aware of the risks, benefits and alternatives to participating in clinical research. This information is provided in the form of a written patient information sheet (PIS) given along with a consent form. Understanding of the written word is based on reading age. This is a reflection on the number of school years a patient must have to understand the text they are reading. Readability scores are a reflection of reading ease and use sentence length and number of syllables to produce a score. PISs should have a target reading age of 13, and a readability score above 60. Aim. The study formally investigated the readability of PISs as an indication of the ability of the PIS to facilitate informed consent. Methods. The PISs for all clinical trials that were open for recruitment during November 2006 to haematology patients from the Western Infirmary, Glasgow, were considered for the study. For ease of analysis only those trials with electronic copies of the PIS were included. Readability scores and reading ages were generated for each information sheet based on the established Flesch-Kincaid Calculation using a Microsoft Word function. Results. Twelve studies were eligible for consideration. Of the 12 studies, 9 were MRC, 1 BNLI, 1 SNLG, and 1 UKMF. The average reading age was 15.8 (range 14.7-16.7) and the average reading ease score was 51.1 (range 44.6-55.1). Conclusions and Summary. All of the PISs were above the recommended reading age (13) and failed to reach the target readability score (>60). These results demonstrate that written information given prior to obtaining consent for clinical trials is too complicated for patients to easily read and understand. This raises important ethical questions in terms of obtaining truly informed consent. We suggest that a) the reading age of the PIS is calculated before submission to the ethics committee, b) that the reading age of the PIS is adjusted to fit the recommended criteria as mentioned above, and c) that the reading age and readability score of the PIS is available to the ethics committee at the time of submission. 0763 EPIDEMIOLOGY, FEATURES AND OUTCOME OF BLEEDING IN PATIENTS WITH ADVANCED HAEMATOLOGICAL MALIGNANCIES FOLLOWED AT HOME: AN ITALIAN SURVEY C. Cartoni, 1 P. Niscola, 2 G.A. Brunetti, 1 G.M. D'Elia, 1 C. Romani, 3 M. Giovannini, 4 L. Cupelli, 4 A. Tendas, 4 D. Piccioni, 2 P. De Fabritiis, 4 R. Foà, 1 F. Mandelli1 1 La Sapienza University, ROME; 2 Sant'Eugenio Hospital, ROME; 3 Armando Businco Cancer Center, CAGLIARI; 4 Tor Vergata University, ROME, Italy Background. Patients with haematological malignancies in advanced phase of disease, experience a wide range of symptoms including major bleeding, which can heavily afflict the quality of life (QoL) and represent a difficult problem solving in the home care (HC) management. Aims. In order to address this issue, a prospective study in a haematological HC setting has been conducted and the results are hereby reported. Methods. Regularly symptom assessment and clinical complications, including haemorrhagic episodes, was recorded. Laboratory evaluation, including complete bloods counts, were frequently executed (3687 blood collections performed during 33.698 home care days, 0.11 blood collection/ day / patient). Patients were followed at home until death for a mean period of 72+144 (1-1132) days. Out of 33.698 days of HC, in 7018 (21%), we recorded a platelet count
uncontrolled massive bleeding affected patients perceived to be close to death and for whom resuscitation is not appropriate, palliative sedation was adopted, if consent was obtained. Results. Out of 469 patients, 123 (26%) developed 232 major bleeding episodes. Severe thrombocytopenia, younger age, diagnoses of acute leukaemias, active infections and mucositis, were significantly correlated with the occurrence of bleeding. Out of 232 major bleedings, 210 (91%) occurred in patients with severe thrombocytopenia. This finding occurred most frequently in patients with acute myeloblastyc leukaemia (AML), acute lymphoblastyc leukaemia (ALL) and blastic crises than in those affected by others haemophaties (p
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haematologica the hematology journa
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Information for readers, authors an
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EHA Executive Board E. Hellström-L
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Poster session I POSTER SESSION POS
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12 th Congress of the European Hema
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dasatinib. Methods. We studied Ikar
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Expression of the oncogene H-Ras an
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is the gene for the erythropoietin
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safety of a slow-release liposomal
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0029 OUTCOME OF THE TREATMENT OF AD
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drug-induced apoptotic response of
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41% in the PI3K- group (p=0.001). I
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Acute myeloid leukemia - Clinical I
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to 60 years (n=116, or 72%) receive
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0056 PROGNOSTIC SIGNIFICANCE OF FLT
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Anemia and Bone marrow failure 0061
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tified with the current protocol. S
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new cases of lead poisoning due to
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icance, from baseline to week 6 (p
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0086 THROMBELASTOGRAPHIC CHART RELI
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trials. The aim of this retrospecti
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tant function in this disease, nega
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ed to a favorable outcome. Bialleli
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stronger levels of p21 in the cell
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hematological centers in one countr
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ure 1). Conclusions. Results for re
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patients. After a median follow-up
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Cytogenetics and Molecular diagnost
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0135 ROUTINE DETECTION OF CYTOGENET
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(MMolR, defined as a BCR-ABL x 100
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0146 ARSENIC TRIOXIDE INDUCES ACCUM
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tinguish between genotypic B-cell l
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Genomics and proteomics 0158 PLASMA
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0164 EVALUATION OF MULTIPLEX LIGATI
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each patient’s replicate conditio
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0174 RELATION BETWEEN LOW PML-RARα
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0179 ESHAP VS GIN AS SALVAGE AND MO
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Immunology and gene therapy 0184 EA
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Cell viability was not affected by
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month is not relevant to chronic Gv
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Infection and supportive care I 020
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0206 POTENTIAL ROLE OF CMV IN THE O
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3H-thymidine uptake in cell culture
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0217 TUBERCULOSIS AMONG A COHORT OF
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0222 COMPARISON OF IWG 2006 AND IWG
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tem (IPSS) to h-MDS was also invest
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PCH97-19) were studied. Conventiona
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of erythroid colonies was reduced i
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0245 POTENT AND SELECTIVE INHIBITIO
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0250 INACTIVATION OF SUPPRESSOR OF
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Bortezomib 1.3 mg/m 2 days 1,4,8,11
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Response was defined according to E
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G-CSF can be used in neutropenic pa
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ence and functional activity of CD8
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itating tumor development, or engag
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phoma and SNT-13, -15 were establis
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usage (2/20 ie 10%) were observed.
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0294 MYCOSIS FUNGOIDES. IMMUNOHYSTO
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(range, 1-6) and 11 treatment cycle
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0306 RISK-ADAPTED IMMUNOCHEMOTHERAP
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functional activity was confirmed b
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No major toxicity, neither hematolo
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enewal potential of X-RAR-positive
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(50-99) respectively. Serial analys
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cell-interaction, adhesion and acti
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0340 ALTERED FIBRIN CLOT STRUCTURE
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Conclusions. This study demonstrate
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0354 FACTOR V LEIDEN HOMOZYGOUS GEN
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Results. From July 2005 through Mar
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0364 CLINICAL EFFICACY OF OXALIPLAT
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one marrow and peripheral blood ste
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ecently suggested (Boissel et al.,
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0378 SAFETY AND EFFICACY OF THE TER
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Cytogenetics and molecular diagnost
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0385 CYTOPLASMIC MUTATED NUCLEOPHOS
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0390 ELTROMBOPAG RAISES PLATELET CO
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factor for the achievement of CR wa
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The cases of RAS showed two peaks o
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is 85%. Seven out of the 25 relapse
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0409 FRACTIONATED RADIOIMMUNOTHERAP
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0414 COMPARATIVE ANALYSIS OF THE CO
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successfully managed with GM-CSF di
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49% for PCR positive patients (95%
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+8 (1 PR+1 HI) and 14/24 pts with c
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(e.g. bcr-abl leading to CML). CSC
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0438 TERC MUTATIONS ANALYSIS IN PAT
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observed in all mice. Analysis of l
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ex-vivo expansion of HUCB-derived H
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ic kidney disease in univariate or
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One hundred eleven CN AML patients,
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to asses intestinal epithelial dama
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genesis of acute myeloid leukaemia
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polymorphism at nucleotide 4889 of
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expression along with a decreased C
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0487 MUTATIONAL STATUS OF NUCLEOPHO
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previously reported, a single cours
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0497 SINGLE AGENT CLORETAZINE (VNP4
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ly received melphalan-based chemoth
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were similar among the 3 groups. Ho
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Methods. Several read-out systems w
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0518 SERUM AND CELLULAR EXPRESSION
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0523 DNA REPAIR INHIBITION IN RESTO
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held true when BMI-1 expression was
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Ph + cells in the bone marrow). We
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derivative chromosome (4p16, 7p14,
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0545 STABILIZED BONE MARROW IS NOT
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obtained in low (Sokal) risk patien
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median dose intensity being 800 (21
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from pivotal clinical trials. Metho
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Cytogenetics and Molecular diagnost
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to set up and optimize a D-HPLC-bas
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0576 WESTERN BLOT IDENTIFICATION OF
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Basic disease was AML (n=5), ALL (n
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0588 EXTRACORPOREAL PHOTOPHORESIS F
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acquire a cytolytic capacity agains
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informed vignettes describing healt
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using CHOP-21 in the UK, pegfilgras
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0609 SOFT TISSUE COMPLICATIONS IN P
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inding lectin (MBL) gene by polymer
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all infection rate (p=0.12) as well
Page 241 and 242: Myelodysplastic syndromes II 0623 M
Page 243 and 244: formation (in total 28 samples). Ti
Page 245 and 246: sion. In addition, confocal analysi
Page 247 and 248: Myeloproliferative disorders - Clin
Page 249 and 250: open-label, multi-centre study enro
Page 251 and 252: iopsies after 6 and 12 months treat
Page 253 and 254: Myeloproliferative disorders Chroni
Page 255 and 256: ash, muscolar cramps, diarrhoea, he
Page 257 and 258: induced significant apoptosis (mean
Page 259 and 260: Myeloma and other monoclonal gammop
Page 261 and 262: the therapy of choice for POEMS is
Page 263 and 264: er patient does not indicate such t
Page 265 and 266: Myeloma and other monoclonal gammop
Page 267 and 268: secutive patients with MM, referred
Page 269 and 270: whether gene expression values may
Page 271 and 272: 0705 THE SHIFT OF TREATMENT FOR NAS
Page 273 and 274: 0710 CLINICO-PATHOLOGICAL FEATURES,
Page 275 and 276: patients presented a stage IV disea
Page 277 and 278: plete remission or recurrent diseas
Page 279 and 280: known at NHL diagnosis, were includ
Page 281 and 282: 0731 THE IMPACT OF HIV ON NON-HODGK
Page 283 and 284: patients had died of their underlyi
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Page 287 and 288: modulated after 24 h (37 up- and 59
Page 289 and 290: 0753 A SUSTAINED REMISSION OF IDIOP
Page 291: and treatment, has rarely been syst
Page 295 and 296: Results. A total of 396 patients we
Page 297 and 298: C30 questionnaire, and the correspo
Page 299 and 300: wave length of light of reflected r
Page 301 and 302: 0783 PREVALENCE OF MEMBRANE PROTEIN
Page 303 and 304: erozygous mother has a more severe
Page 305 and 306: 0794 CARDIAC MANIFESTATIONS IN A BR
Page 307 and 308: 0799 INEFFECTIVE ERYTHROPOIESIS IN
Page 309 and 310: p=0.014 and p=0.003, respectively).
Page 311 and 312: 0809 CURRENT APPROACH TO CHELATION
Page 313 and 314: Thrombosis II 0814 UNSUSPECTED PULM
Page 315 and 316: the 97.5th percentile (5.2 U/mL) ge
Page 317 and 318: symptoms of DVT, 3 (7.89%) previous
Page 319 and 320: Transfusion medicine and vascular b
Page 321 and 322: tions (most bacterial, 2 malaria, 6
Page 323 and 324: 0844 INCREASED LEUKOCYTE-PLATELET I
Page 325 and 326: 0851 CORD BLOOD DERIVED MESENCHYMAL
Page 327 and 328: SIMULTANEOUS SESSION II Chronic mye
Page 329 and 330: 0862 COMPARISON OF DASATINIB TO HIG
Page 331 and 332: 0867 COMPREHENSIVE GERIATRIC ASSESS
Page 333 and 334: 0872 MN1 INDUCES LEUKEMIA IN MICE A
Page 335 and 336: 0877 PAX5/TEL TRANSDUCED PRE-BI CEL
Page 337 and 338: 0882 CISPLATIN INDUCES THROMBIN GEN
Page 339 and 340: have an early manifestation of typi
Page 341 and 342: 0892 INTEGRATION OF GENOME WIDE SNP
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0897 THE PHENOTYPE OF JAK2V617F MUT
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gest diverse sequences of NPM mutan
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2004 to January, 2006. At enrollmen
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with a p value of ≥ 0.10. Sets of
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BRIC 126 and 4N1K) in cells lacking
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sclerosis[NS] and 12 Mixed cellular
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0927 MK-0457, A NOVEL MULTIKINASE I
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Publication Only 0933 CLINICAL FEAT
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HSCT from the available Asian as we
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that the incidence of JAK2 mutation
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without type 2 diabetes. Methods. T
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pts (38.8%) that were isolated (abs
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y using the Miltenyi CD34 isolation
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0969 COMPARISON OF CD25 IMMUNOHISTO
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cytes was 24 days (range 8-32 days)
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a cytogenetic hallmark for M4/M5 su
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normal human BM B progenitor cells
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0994 INCIDENCE OF INVASIVE ASPERGIL
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3 of disease relapse.TRM at day+100
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survival of five years. This dismal
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1011 FLOW CYTOMETRIC DNA INDEX AND
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1018 THE EFFECT OF THE SUGARBAKER P
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1024 KIR/HLA CLASS I MISMATCHING AN
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ment details and thrombotic histori
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1036 INCIDENCE AND SPECTRUM OF INFE
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tinely by our stem cell lab prior t
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tion to a translocation t(5;14)(q35
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ment of CML and induces a high rate
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due to reactivation and/or progress
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1063 ABNORMAL METHYLATION STATUS OF
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1069 CLINICAL RELEVANCE OF REGULATO
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1076 SERUM LEVELS OF SOLUBLE HLA CL
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patient is still undergoing intensi
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nificant MVD differences were detec
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dictor of OS (p=0.004). High dose t
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1099 ALTERATIONS IN THE NATURAL KIL
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1105 CYTOGENETIC ABNORMALITIES IN 3
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1110 CONSTITUTIVE ACTIVATION OF STA
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efficacy results with a well-tolera
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unmutated VH genes, and high and lo
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Aims. Aim of this study was to pred
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tested across a wide range of human
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were incidentally diagnosed (52%).
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ß2GP1 may be considered as determi
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characterized in 198 β thalassaemi
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1155 PLATELET AGGREGATION IN CHILDR
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to-pelvic or perineal trauma. No me
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in age. High prevalence of LBM amon
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ecause some of the patients were or
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of the drug is crucial to allow lon
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egarding cost analysis of ASCT in G
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ID Allo-BMT, 1 family one mismatche
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admitted to ICU were discharged des
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events -Postoperative states: 9,19%
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efficacy and decreased atherosclero
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1217 INCIDENCE OF EARLY STAGE IDIOP
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1691GA and 1 homozygous 1691AA. The
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1230 ASSOCIATION OF HEPARANASE GENE
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posed, was: DF = (CD43%+FMC7%+CD79b
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treatment of acute promyelocityc le
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loaded group and 35 (70%) were non-
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mild. Fas and soluble Fas ligand le
Page 467 and 468:
1265 POSACONAZOLE THERAPY IN REFRAC
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ly express the cytoplasmic (inactiv
Page 471 and 472:
findings confirmed the significant
Page 473 and 474:
a less favorable prognosis. Interes
Page 475 and 476:
disease (HD), 4 patients (9%) with
Page 477 and 478:
1295 HEMOPHAGOCYTIC LYMPHOHYSTIOCYT
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phamide 750 mg/m 2 on day 1, vincri
Page 481 and 482:
nomenon is unclear. It has been sug
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oped mild thrombocytopenia (platele
Page 485 and 486:
gle dose of 54mg/m 2 rituximab furt
Page 487 and 488:
patients (pts), 36 male, with acute
Page 489 and 490:
esulting alterations of the endothe
Page 491 and 492:
(range 1-7) and 28,6% of patients h
Page 493 and 494:
three decades. In vivo, Ara-C is tr
Page 495 and 496:
statistical analysis. Results. Seve
Page 497 and 498:
Results. Though there was no recogn
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2% and 19% of patients with or with
Page 501 and 502:
were older than 65 y) which can be
Page 503 and 504:
1376 RESVERATROL INDUCED P53 MEDIAT
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median time of 21 days to reach >0.
Page 507 and 508:
ly. Conclusions. 1. Combined intens
Page 509 and 510:
to the presence of IVS1-6 mutation
Page 511 and 512:
fy predictive factors for these abn
Page 513 and 514:
acute leukemia. However, we cannot
Page 515 and 516:
agents. They involve primarily the
Page 517 and 518:
voriconazole for 2 months followed
Page 519 and 520:
typed using a commercially availabl
Page 521 and 522:
low up of ABL KD mutations’ level
Page 523 and 524:
with development of BC/AP. 2. EVI-1
Page 525 and 526:
1447 THE IMPACT OF DISPARITY IN SHO
Page 527 and 528:
three had visual field defects, two
Page 529 and 530:
acquired mutation most prone to cau
Page 531 and 532:
1466 ROS GENERATION AND APOPTOSIS I
Page 533 and 534:
1473 DIARRHEIC SYNDROME, A CLINICAL
Page 535 and 536:
gene fusion is an independent progn
Page 537 and 538:
1484 EPIDEMIOLOGY AND RESPONSE TO T
Page 539 and 540:
1492 FREQUENCY OF MEDITERRANEAN GLU
Page 541 and 542:
immune globulin was administered at
Page 543 and 544:
maintained on Imatinib 400 mg. Afte
Page 545 and 546:
Hodgkin’s disease is well known,
Page 547 and 548:
gram provided a unique chance to de
Page 549 and 550:
even when ADP-induced plt activatio
Page 551 and 552:
medullary involvement of multiple m
Page 553 and 554:
1540 ADMINISTRATION OF G-CSF AND CH
Page 555 and 556:
1548 QUALITY ANALYSIS OF THE MULTID
Page 557 and 558:
Index of authors A Aapro, M., 0377,
Page 559 and 560:
Bahr, J., 0148 Bai, M., 1551 Baia,
Page 561 and 562:
Bottini, P.V., 1181 Botto, B., 0408
Page 563 and 564:
Chaidos, A., 0456, 0498, 0599, 0686
Page 565 and 566:
De Keersmaecker, K., 0442, 0875 De
Page 567 and 568:
El-Sharkawy, N., 0016 Elwahidi, G.,
Page 569 and 570:
Garcia-Frade, J., 0346, 0680, 1105
Page 571 and 572:
H Haas, N., 0742 Haas, O.A., 0001,
Page 573 and 574:
Jaksic, B., 0127, 0446 Jaksic, O.,
Page 575 and 576:
Körmöczi, G., 0848 Kornblau, S.,
Page 577 and 578:
Lin, L.-I., 0030, 0484 Lin, T., 012
Page 579 and 580:
Massé, A., 0249 Massó, P., 1287,
Page 581 and 582:
Musto, P., 0254, 0401, 0422, 0569,
Page 583 and 584:
Papadavid, E., 1442 Papageorgiou, E
Page 585 and 586:
Probatova, N., 0704 Prochazka, V.,
Page 587 and 588:
Rykavicin, O., 0504 Ryningen, A., 0
Page 589 and 590:
Sirard, C., 0127, 0128 Sirigou, A.,
Page 591 and 592:
Thiebaut, A., 0454 Thieblemont, C.,
Page 593 and 594:
Vincenzi, C., 1060 Viniou, N.-A., 0
Page 595 and 596:
Zouabi, H., 0503, 0999 Zoumbos, N.C
Page 597 and 598:
Page 599 and 600:
Page 601 and 602:
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