bluuebunny/crossref_metadata_2025 huggingface dataset:
PB2219 EFFECTS OF SYSTEMIC MASTOCYTOSIS ON BONE HEALTH IN SOUTH‐EAST SCOTLAND
Authors: Peters N. , Teh C.‐H. , Seth S.
Published: June 2019

Background:

Systemic mastocytosis (SM) is a rare condition characterised by excess mast cell production and often involves multiple organs. One organ system becoming increasingly recognised as being affected by systemic mastocytosis is the skeletal system, with reports of increased prevalence of osteoporosis, osteopenia and osteosclerosis represening the main findings.

Aims:

We aimed to assess the extent of bony involvement of SM in patients in our area, South‐East Scotland who had been referred to the Haematology services in NHS Lothian in order to help guide local policies regarding assessment of bone health upon a diagnosis of systemic mastocytosis.

Methods:

Clinical records of patients with a diagnosis of SM referred to our services in NHS Lothian available on our local TRAK system were interrogated to investigate for the presence of osteoporosis, osteopenia, osteosclerosis. Further, where available, DEXA bone scan results were tabulated.

Results:

We identified 18 patients with a diagnosis of SM with multiple variants identified including indolent (15/18), cutaneous, Aggressive with myelofibrosis (1/18) and SM with eosinophilia (1/18) and unclassified (1/18). It was found that 12/18 patients had evidence of osteoporosis (6/18), osteopenia (5/18) or osteosclerosis (1/18) representing a significant disease burden. Further, 13 of the 18 patients had a DEXA scan meaning there is a large portion of patients not being investigated routinely for bony involvement. The average age of patients with osteoporosis and SM was 60 years old with a range from 41 to 74, suggesting a profound effect on bone health at an early age.

Summary/Conclusion:

We have identified a significant proportion of patients with SM having bony involvement. It has been suggested that DEXA scans form part of initial workup of these patients upon diagnosis, with bisphosphonates used as a first line treatment. The findings of our study support the proactive investigation of bone health in SM.
PB2220 SURVIVAL OUTCOME OF HIGH‐RISK PATIENTS WITH MYELOFIBROSIS TREATED WITH ALLOGENEIC HEMATOPOIETIC CELL TRANSPLANTATION FOLLOWING REDUCED‐INTENSITY CONDITIONING REGIMENS
Authors: Lee J. , Yoon J.‐H. , Yoon S. Y. , Kim C. , Park S.‐S. , Kim H.‐J. , Lee J. W. , Lee S. , Eom K.‐S.
Published: June 2019

Background:

Allogeneic hematopoietic cell transplantation (allo‐HCT) is the only curative treatment for myelofibrosis (MF), but substantial risk of transplantation‐related mortality (TRM) is a considerable obstacle especially in elderly patients or in patients with severe comorbidity. In the era of Janus kinase (JAK) inhibitors, transplantation may be delayed in good responders but cytopenia is a major complication, and we confront refractory patients who should be treated with allo‐HCT immediately.

Aims:

Here, we are going to show our experiences on high‐risk MF patients who were treated with reduced‐intensity conditioning (RIC) allo‐HCT.

Methods:

Here, we are going to show our experiences on high‐risk MF patients who were treated with reduced‐intensity conditioning (RIC) allo‐HCT.

Results:

All showed successful engraftment. Including 2 patients who were conditioned with nonmyeloablative regimen, total 6 (15.8%) showed delayed graft failure at a median 8 months post‐HCT (range, 3.8‐13.8). Two recovered after stem cell booster from the same donor, other 2 received donor lymphocyte infusion but showed progression, and 1 died after 2nd allo‐HCT. Among the 33 patients with post‐HCT bone marrow (BM) exam results, 29 (87.8%) showed significant improvement of BM fibrosis (range, 14‐661 days). After median follow‐up duration of 24.6 months (range, 6.1‐124.8), 2‐year OS was 64.6% (64.3% in triple‐negative subgroup), 2‐year nonrelapse mortality was 27.4%, and 2‐year graft‐and‐relapse‐free survival (GRFS) was 28.4%, which suggested there were fatal high‐grade GVHD in this cohort. Acute GVHD was observed in 18 patients (5 grade II, 7 grade III, 4 grade IV) and a cumulative incidence of grade III‐IV acute GVHD was 30.6%. Chronic GVHD was observed in 13 (4 mild, 2 moderate, 7 severe), and a cumulative incidence of severe chronic GVHD was 27.5%. Among them, severe hepatic GVHD was observed in 9 patients (5 acute, 4 chronic). Among 11 dead patients, 7 died due to fatal hepatic GVHD, 1 died of lung GVHD, and 3 died due to septic pneumonia. Multivariate analysis revealed 2‐year OS was significantly poorer in patients who suffered from hepatic GVHD (HR = 9.8, 95%CI 1.0‐94.7, p = 0.022) and the significant cause of fatal hepatic GVHD was unrelated or mismatched donor HCT.

Summary/Conclusion:

RIC allo‐HCT can be a valid choice providing graft‐versus‐myelofibrosis effect for advanced and triple‐negative MF patients, but fatal hepatic GVHD was frequently observed especially in unrelated or mismatched donor transplants which should be overcome in the future.
PB2242 COMPARISON BETWEEN MANUAL AND AUTOMATED PLATELET COUNTS OF ADULT PATIENTS WITH THROMBOCYTOPENIA AT ST. PAUL'S HOSPITAL ILOILO (SPHI), PHILIPPINES
Authors: Castromayor J. , Cadete L. Agreda‐
Published: June 2019

Background:

Platelets are more difficult to count compared to other blood cells. Platelet counting is a significant tool used in clinical investigation to prevent bleeding complications. There is a general agreement that automation bring precision and accuracy for platelet counts in healthy individuals. Automation of platelet counting has been embraced in most part of the world, particularly in more developed countries. However, manual platelet counting & estimation is still being done by skilled medical technologist to generate platelet results in most developing countries like ours. The method of platelet counting in thrombocytopenic patients is still controversial.

Aims:

To compare if there is significant difference between automated and manual platelet counts of adult patients with thrombocytopenia at the clinical laboratory of Saint Paul Hospital Iloilo City, Philippines.

Methods:

This is a retrospective‐analytical study of 384 samples of complete blood count (CBC) results with thrombocytopenia that contain both automated and manual platelet count values.

Results:

Out of the 384 adult patients with thrombocytopenia, there was an approximately 1:1 ratio based on sex. Thrombocytopenia was most common between 6th to 8th decades of life affecting 36% of patients. The mean of the automated platelet levels was approximately 76 × 109/L with the standard deviation of 45; while the manual platelet count results was 170 × 109/L with the standard deviation of 99. Among adult patients with thrombocytopenia, there was a significant difference between manual and automated platelet count results with p value < 0.05).

Summary/Conclusion:

Thrombocytopenia affect both male and female sex equally. The higher the age group, the higher is the incidence of thrombocytopenia. Manual platelet counts are highly variable compared to the automated values. There was a significant difference between manual and automated platelet count results.
PB2246 CLINICAL FEATURES AND TREATMENT OUTCOMES OF IMMUNE THROMBOCYTOPENIC PURPURA IN INFANTS; A SINGLE CENTER RETROSPECTIVE STUDY
Authors: Park K. M. , Yang E. J. , Lim Y. T.
Published: June 2019

Background:

Immune thrombocytopenic purpura (ITP) in children less than one year of age is less well characterized compared to ITP in toddlers and school‐age children.

Aims:

Since children of different ages may have differing clinical courses, better delination of the natural history of ITP in infants is needed.

Methods:

We retrospectively reviewed the admission records of 248 consecutive pediatric patients between 1 month and 15 year of age admitted and treated for acute ITP at Pusan National University Children's Hospital from 2009 through 2017. All of these patients less than 1 year of age were identified and enrolled. We investigated their demographics, clinical features, laboratory examinations, respond to treatment and long‐term outcomes and compared to those of children 1 to 10 years of age.

Results:

Ninety nine infants were identified. Male to female ratio was highest in infants and decreased with age. Seventy nine (79.8%) of the 99 infant were found to be under 6 months old. The median platelet counts at diagnosis was 6 x 109/L (0∼76). Minor bleeding (bleeding score 0‐2) was significantly dominant in infant compared to older ages. Eighty two out of 85 patients (96.5%) achieved complete remission after initial intravenous immunoglobulin(IVIG) treatment. The relapse rate after initial CR was significantly lower than older ages (p = 0.003). The platelet count after IVIG treatment in infant showed more rapid rising compared to older ages (p = 0.02), and the mean time to platelet counts above 100x109/L was 6.4+4.5 days. Follow up information at 6 months was available for 85 infants. Chronic ITP at 6 month was seen less frequently in infants (4.7%) than in children 1 to 10 years of age. (23.2%. P < 0.01).

Summary/Conclusion:

Infants with acute ITP respond more favorably to IVIG treatment and are less likely to develop chronic ITP compared to children 1 to 10 years of age.
PB2255 ADULT ITP PATIENTS TREATED WITH ROMIPLOSTIM IN DAILY CLINICAL PRACTICE: SINGLE CENTER EXPERIENCE
Authors: Janssens A. , Vergote V. , Schoemans H. , Beckers M. , Devos T. , Dierickx D. , Delforge M. , Maertens J. , Verhoef G. , Vandenberghe P.
Published: June 2019

Background:

Since May 2007 we can treat in Belgium adult primary immune thrombocytopenia (ITP) patients with the TPO receptor agonist (TPO‐RA) romiplostim (clinical trials, reimbursed, medical need).

Aims:

We wondered what the baseline and treatment characteristics of this ITP patient population was.

Methods:

From May 2007 till May 2018, 60 adult ITP patients started treatment with romiplostim at the University Hospitals of Leuven.

Results:

The baseline characteristics of this cohort are as follows: 35 of 60 patients were male; diagnosis of primary ITP was made in 42 patients; the 17 patients with secondary ITP had a history of lymphoma (n = 6), Evans syndrome (n = 5), a previous liver or renal transplant (n = 3) or an underlying inherited disorder (n = 3); mean age and platelet count at ITP diagnosis was 53years (y) (range 1‐90y) and 16000/ml; median time between ITP diagnosis and the start of romiplostim was 60months (mo) (range 0‐492mo); at start of romiplostim 7 patients fulfilled the definition of acute, 7 of persistent and 45 of chronic ITP; median platelet count at start of the drug was 20000/ml; 32% (19/59) were splenectomised and 39% (23/59) needed 3 3 ITP treatments (range 3‐8) before the start of the TPO‐RA.

At 30 november 2018, median time on romiplostim was 21.5mo (range 2weeks ‐10.5y); treatment was ongoing in 38% (n = 23/60) of patients; reasons for discontinuation was non‐ITP related death (n = 17), ITP related death (n = 1), treatment‐free remission (TFR) (n = 10), patient's decision followed by TFR (n = 2), splenectomy followed by TFR (n = 2), doctor‘s decision (n = 1), loss of response (n = 2) and loss of follow up (n = 1). Median dose of romiplostim needed for a safe platelet count was 3.75 m/kg.

Summary/Conclusion:

These data show that treatment with romiplostim is not “for all” forever. Non‐ITP related death (median time on drug 20.5mo) and TFR (median time on drug 5mo) were the most common reasons of discontinuation of the drug.
PB2256 DISSOLVED PSEUDO‐THROMBOCYTOPENIA BY USING EVIDENCE‐BASED RESEARCH
Authors: Chen S. H. , suen T.
Published: June 2019

Background:

Platelet clumping is a common laboratory phenomenon that causes pseudo‐thrombocytopenia (PTC), spurious thrombocytopenia due to persistence of antibody or agglutinins. Microscopic examination of the peripheral blood smear is of key importance to confirm the diagnosis and to avoid any inappropriate or dangerous clinical and therapeutic decision. How to dissolve platelet clumping and make sure correct platelet count is an important procedure. Ethylenediaminetetraacetic acid (EDTA) is a well‐known anticoagulant for the hematological tests (CBC). EDTA‐dependent pseudo‐thrombocytopenia is the most common causes of platelet clumping in medical laboratory.

Aims:

To dissolved platelet clumping ex vivo and correct platelet count in medical laboratory avoid further inappropriate treatment and waste of medical resource.

Methods:

We drew blood of the patient in three tubes containing three different types of anticoagulants; EDTA, Sodium Citrate and Heparin. The three blood samples were run in fully automated Hematology analyzer (SYSMEX XN‐3000).Three tubes also process in water bath at 37 °C 10 minute and vortex until run automated Hematology analyzer again.

Results:

54 years old female patient was referred to our lab for investigations as a case of implicated thrombocytopenia. The CBC revealed normal parameters apart from a low platelet count; 35 x10^9/L. A blood smear was ordered and it revealed server platelet clumping. Using different types anticoagulants, EDTA, Citrate, heparin result similar platelet count and still reveal platelet clumping on blood smear. We further pre‐treated warm water bath(37 °C) plus vortex before analysis. Platelet count was counted 138 x10^9/L by automatic hematologic analyzer in pre‐warm blood specimen. The smear also checked by microscopy without phenomenon of platelet clumping.

Summary/Conclusion:

Platelet cold agglutinin is confirmed by pre‐warm blood specimen which is a rare cause in pseudo‐thrombocytopenia. We used methods of evidence‐based medicine (EBM) to find out patient with cold agglutinins that affect platelet counting.
PB2262 HEALTHCARE RESOURCE UTILIZATION (HRU) ASSOCIATED WITH MINIMAL RESIDUAL DISEASE (MRD) STATUS IN ADULTS WITH B‐CELL PRECURSOR (BCP) ACUTE LYMPHOBLASTIC LEUKEMIA (ALL)
Authors: Pigneux A. , Montesinos P. , Cong Z. , Zhang X. , Pownell A. K. , Wieffer H. , McKendrick J.
Published: June 2019

Background:

The presence of MRD is the most important risk factor for relapse and mortality in patients with BCP ALL who achieve complete remission (CR). Given the poor outcomes associated with MRD‐positive (MRD+) ALL, achieving MRD‐negativity (MRD‐) is critical. However, the HRU associated with MRD status is unknown.

Aims:

To investigate the HRU associated with MRD status among adult patients with BCP ALL in first CR (CR1) in France, Germany, Italy, Spain and UK (EU5).

Methods:

A cross‐sectional survey of physicians who use MRD testing in adults with BCP ALL in the EU5 countries was used to estimate the HRU. Physicians must be board‐certified or specialized in hematology/hemato‐oncology, have ≥ 5 years’ experience with ALL, have seen ≥ 2 ALL patients in the last 12 months or ≥ 5 patients in the last 5 years, and have experience using MRD testing in clinical practice. Two to 4 physicians were recruited in each country. Each physician completed a web‐based questionnaire and was followed by a telephone interview. Descriptive analyses were conducted. Outpatient visits to healthcare professionals and hospital resource use for ALL patients in CR1 with or without MRD during an average period of 6 months (assuming no relapse or allogeneic hematopoietic stem‐cell transplant occurred) were reported for each country and across countries.

Results:

According to the survey, across the countries, patients with MRD+ ALL had an average of 9.2 (ranging 4∼12) visits to hematologist/oncologist, 1.4 (0∼2.5) visits to radiologist, 1.8 (0∼3) visits to other specialist physician and 2.6 (0∼4.5) visits to general physician during a 6‐months period, compared with 7.4 (1∼11), 1.1 (0∼2.3), 1.4 (0∼2.3) and 2.3 (0∼4.0) visits, respectively, in patients with MRD‐ ALL (Table 1). The average proportion of patients with at least one hospital admission in patients with or without MRD was 64.7% (ranging 40.0%∼83.3%) vs 39.3% (0∼83.3%). Of the patients with at least admission, patients with MRD+ ALL had an average of 3.2 (1.5∼4.3) admissions with a mean length of stay (LOS) of 13.2 (0.5∼19.8) days per admission, compared with 2.1 (0∼4.3) admissions and a mean LOS of 8.9 (0∼15.3) days per admission in patients with MRD‐ ALL (Table 2).

Summary/Conclusion:

From the physicians’ estimates of the HRU for patients with ALL in CR1 in the EU5 countries, presence of MRD+ is generally associated with higher use of outpatient visits to physicians across different specialties and with higher use of hospital resources (except Spain). The results should be interpreted with the limitation of small sample sizes in each country.

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PB2266 HOW TO IMPROVE HEMATOLOGIC REFERRAL? ANALYSIS AND RECOMMENDATIONS
Authors: Saraiva F. , Postolachi R. , Vicente M. I. , de Sousa A. Botelho
Published: June 2019

Background:

Incidental laboratorial abnormalities detected in asymptomatic persons are increasingly becoming a frequent motive for referral to a haematological consultation, overloading outpatient units that are run by small teams. These referrals, along with the subsequent acceptance by the triage manager and further haematological investigation by a haematologist, originate from the fear of missing incipient severe haemopathies.

Aims:

To evaluate the adequacy and the result of these referrals to ascertain the hypothesis that a large proportion of the cases have no indication for specialized haematological care.

Methods:

Retrospective analysis of patients referred to the Haematology consult of a central hospital over a 2‐year period (Jan/16‐Dec/17), excluding the ones who already had a known diagnosis. We evaluated the reason for referral, the final diagnosis and at a second stage we tried to quantify the referrals which could be managed in primary care, and their origin.

Results:

Out of 1162 evaluable patients (236 still in study), the majority (65%) came from Primary Health Care. The motive, in 85% of the cases, consisted in blood count abnormalities (59% cytopenias). In 13% of the cases, the initial abnormality no longer existed upon arrival. Finding an underlying pathology was possible in only 48% of the cases. From these, 14% could have been managed in Primary Health Care (e.g. iron deficiency; vitamin deficits) and 86% were haematologically relevant. As for the referrals which could have been avoided (no underlying haematological pathology or unconfirmed motive) the most frequent causes were cytopenias (anaemia 33%, leukopenia 15%, thrombocytopenia 12%) and they originated from Primary Health Care (72%).

Summary/Conclusion:

In this sample, we confirmed that a high proportion of referrals are manageable in Primary Health Care, particularly cytopenias, part of which are no longer present at the first haematological evaluation. The probability of a haematological investigation resulting in a clinically relevant diagnosis is very low, seemingly not justifying the consult time, the costs for the system or the personal costs for the patient. The elaboration of specifically designed recommendations for Primary Health Care and the implementation of interaction/discussion networks of pre‐triage cases are indispensable measures to optimize the haematological referral process.
PB2282 REASSESSING THE EFFICACY OF LOW DOSE RASBURICASE FOR MANAGEMENT OF TUMOR LYSIS SYNDROME IN HIGH RISK HEMATOLOGIC MALIGNANCIES
Authors: Heering G. , Loebstein R. , Rabinovitch H. Rotem , Avigdor A. , Merkel D. , Benjamini O. , Volchek Y. , Nagler A. , Canaani J.
Published: June 2019

Background:

Tumor lysis syndrome (TLS) is a potentially life‐threatening metabolic derangement seen in aggressive hematologic malignancies which is characterized by the massive release of toxic metabolic byproducts and consequent kidney damage. Whereas rasburicase is an established standard of care for management of TLS, the high cost of administering standard dosing of rasburicase has led to exploring lower doses of rasburicase.

Aims:

Assess our center's experience with lower dosing of rasburicase for management of TLS in aggressive hematologic malignancies.

Methods:

We studied all adult patients with hematologic malignancies who were treated with rasburicase in our institution between the years 2011‐2018. Charts were reviewed and demographic, clinical, and laboratory data were collected. Subjects were defined to be at high risk for TLS if they had any of the following features: acute leukemia, elevated uric acid levels at baseline (>7.5 mg/dL) or aggressive lymphoma.

Results:

Fifty‐five patients were evaluated. The median age of patients was 67 years (range 22‐92) consisting of 20 patients with acute leukemia, 21 with aggressive lymphoma, 11 with multiple myeloma, and 3 with other hematologic cancers. Nearly all analyzed patients (98%) were considered to be at high risk for TLS and 44 patients did meet criteria for clinical TLS. Twenty‐six patients (47%) received allopurinol prophylaxis. The median dose of rasburicase administered was 3 mg (range 1.5‐9) with 10 patients being given a second dose of rasburicase. The median baseline levels of uric acid and creatinine were 13.9 mg/dL (range 6.5‐42.8) and 2.18 mg/dL (range 0.63‐8.15), respectively. At 24 hours post administration of rasburicase the median uric acid levels were 7.9 mg/dL (range 0.8‐17.5) while at 48 hours they were 4.9 mg/dL (range 0.8‐13.5). Notably, 54 patients (98%) experienced a decrease in uric acid levels of at least 10% within the first 24 hours following rasburicase administration and 40% of patients had normal uric levels by that time. Within the initial 24 hours, creatinine levels were significantly improved in only a minority of patients with only 10 patients (18%) experiencing a decrease of more than 10% compared to baseline levels. Of note, 15 patients required dialysis during their admission and 26 patients died during the index hospitalization.

Summary/Conclusion:

The majority of subjects in our analysis of a high‐risk patient population responded favorably to low dose rasburicase in terms of rapid uric acid reduction although rates of dialysis and early mortality were substantial. Our data illustrate and confirm the feasibility of lower dosing of rasburicase to patients with aggressive hematologic malignancies.
PB2284 PSYCHOLOGICAL DISTRESS IN PATIENTS WITH HEMATOLOGICAL MALIGNANCIES: PREVALENCE AND ASSOCIATED FACTORS
Authors: Kacem K. , ghedira H. , sayadi M. , stambouli H. , eddhib J. , m'sadek F. , zriba S.
Published: June 2019

Background:

The diagnosis of hematological malignancy (HM) is associated with psychological suffering for patients who graft on their physical suffering. Psychological distress (PD) affects patient's quality of life and their ability to adhere to treatment.

Aims:

The exact prevalence of this event among patients with HM is not well specified. In this work, it is proposed to determine the prevalence of PD in patients with HM and the factors predicting its occurrence.

Methods:

We conducted a retrospective descriptive study that included 100 patients with HM followed in 2 Clinical Hematology departments in Tunis between 2010 and 2017. The DP was explored by the Distress Thermometer Scale (DTS) and by the validated Arabic version of the Hospital Anxiety and Depression Scale (HADS).

Results:

PD was detected in 40 patients byDTS and 43 patients by HADS.

18 patients had an anxiety disorder alone, 5 patients had depressive disorder alone and 18 had concurrent anxiety and depression.

DTS was significantly correlated with the global HADS, HADS‐depression and HADS‐Anxiety.

In multivariate analysis, the associated comorbidities, the governorate of residence and the lack of complete response after treatment were predictive of PD occurrence.

Summary/Conclusion:

This study has shown a high prevalence of PD among patients with HM. Tunisian hematologists need to know how to detect these disorders as early intervention enhance their psychological wellbeing and improve their disease outcome.
PB2288 BENDAMUSTINE‐BORTEZOMIB‐DEXAMETHASONE (BVD) IN HEAVILY PRETREATED MULTIPLE MYELOMA: OLD/NEW ARM IN NOVEL AGENTS’ WORLD
Authors: Cerchione C. , Martinelli G. , Rocco S. , Palmieri S. , Nappi D. , Pareto A. E. , Pane F. , Catalano L. , Ferrara F.
Published: June 2019

Background:

Bendamustine is a bifunctional alkylating agent, with low toxicity, proved to be effective in relapsed, refractory and in new diagnosed Multiple Myeloma (MM).

Aims:

In this retrospective study, It has been evaluated efficacy and tolerance of Bendamustine, in combination with bortezomib‐dexamethasone (BVD) in patients with relapsed and refractory MM (rrMM), whose prognosis is particularly severe. A regional retrospective real‐life analysis of patients with rrMM who had been treated with BVD as salvage therapy has been performed.

Methods:

56 patients (31 M/25 F), with rrMM, median age at diagnosis 57.3 years (r. 36‐82), median age at start of treatment 61.8 years (r.37‐83) treated with several lines of treatments (median 6, r. 2‐11), every refractory to all the drugs previously received (also Bortezomib), received BVD (Bendamustine 90 mg/sqm days 1,2; Bortezomib 1.3 mg/sqm days 1,4,8,11, Dexamethasone 20 mg days 1,2,4,5,8,9,11,12, Pegfilgrastim day +4) every 28 days, until progression.

ISS was equally distributed, and cytogenetic was evaluable in 12 patients, and in particular one del13q and one t (11;14). All the patients had previously been treated with schedule containing bortezomib and IMIDs, and 30% had also received radiotherapy. 67% of them had undergone at least to a single auSCT. All patients were relapsed and refractory to last therapies received before BVD.

Results:

Bendamustine was well tolerated, with grade 3 transfusion‐dependent anemia in 41% of patients, and 37% grade 3 neutropenia (no ospedalization was required, no septic shocks were observed). No severe extrahematologic toxicity was observed, only grade 1 gastrointestinal side effect (nausea), treated by common antiemetic drugs.

According to IMWG, after a median follow‐up of 14 months (r.2‐36), ORR was 64% (36/56: 4 CR, 7 VGPR, 16 PR, 9 MR) with 8 PD and 12 patients in SD, which can be considered as an impressive result in this subset of rrMM patients. In particular, for 11 patients, BVD was, after having achieved at least a PR, a bridge to second auSCT, and for two patients a bridge to alloSCT. Three patients have shown a notable PR after failure of novel agents (i.e. Carfilzomib and Pomalidomide).

Median time to response was 1.2 months (r.1‐3), median OS from diagnosis was 62.7 months (range 6‐151), median OS from start of Bendamustine was 9.8 months (range 2‐36).

Summary/Conclusion:

The triplet Bendamustine‐Bortezomib‐Dexamethasone has shown significant efficacy in a particularly severe setting of patients, relapsed and refractory to all available therapeutic resources, and, in particular cases, it could be considered as a bridge to a second autologous or allogenic SCT.
PB2289 HEALTH‐RELATED QUALITY OF LIFE AND OXYGEN TRANSPORT IN PATIENTS WITH NEPHROGENIC ANEMIA
Authors: Bondarchuk S. , Kovalev A. , Nikolay R.
Published: June 2019

Background:

End‐stage renal disease (EsRD) is often associated with an anemia. Most patients retrieve erythropoiesis stimulating agents, and so they presents with mild anemia. The tolerance of anemia of each patient is individual. Determination of venous blood gas (VBG) in conjunction with study of health‐related quality of life (HRQoL) can help in the individualization of treatment.

Aims:

To investigate the relationship between health‐related quality of life and venous blood gas in patients with nephrogenic anemia.

Methods:

30 patients with EsRD undergoing hemodialysis and receiving erythropoes stimulating therapy were involved in the study. 20 men, median age 52 years. Polycystic kidney disease was caused by the development of EsRD in 7 (23%) patients, diabetic nephropathy in 6 (20%) patients, glomerulonephritis also in 6 (20%) patients, nephrosclerosis in 5 (17%), and other causes in the remaining 6 patients (20%). HRQoL was studied using the FACT (Functional Assessment of Cancer Therapy Scale) questionnaire. Indicators of complete blood count, VBG were evaluated.

Results:

The hemoglobin median in the studied patients showed 105 g/l (81‐112 g/l), 26 patients experienced mild anemia (87%), 3 patients (10%) ‐ moderate anemia. in 1 patient there was no anemic syndrome. SvO2 median is 62.3%, while in only 18 (60%) patients the level of SvO2 is ≥60%. In general, the results of HRQoL analysis showed its deterioration in patients on all scales of the FACT questionnaire; the highest score was obtained in the scales “Physical well‐being” (Me 11 points) and “Anemia” (Me 30 points). When conducting a correlation analysis of hemoglobin and HRQoL, a statistically significant relationship was found on the scales “Physical well‐being” (r = ‐0.45; p < 0.05), “Emotional well‐being” (r = ‐0.39 p < 0, 05) and Anemia (r = ‐0.51; p < 0.05). A statistically significant correlation was obtained between the level of hemoglobin and the level of oxygen in venous blood. At the same time, no significant correlation was established between HRQoL and the saturation of venous blood (p > 0.2).

Summary/Conclusion:

According to the results of the HRQoL analysis, it was established that in patients with nephrogenic anemia, HRQoL decreases depending on the concentration of hemoglobin. The generally accepted target level of hemoglobin (100‐110 g/l) allows to avoid severe complications, but not in all cases it can adequately meet the need for O2 in patients. According to the data obtained, only 60% of patients achieved SvO2 ≥ 60%, which indicates the presence of tissue hypoxia.
PB2313 OCULAR MANIFESTATIONS OF SICKLE CELL DISEASE: A SINGLE CENTRE STUDY
Authors: Efobi C. , ejiofor O. , uzozie C. , onyiaorah A.
Published: June 2019

Background:

Sickle cell disease (SCD) is an inherited haemolytic disorder that affects virtually all body systems including the eye. Ocular complications of SCD could have dire consequences if not detected early.

Aims:

We aimed at determining the prevalence of ocular complications of SCD in our patients.

Methods:

This study was carried out on patients attending both the adult and Paediatric Sickle cell clinic at Chukwuemeka Odumegwu Ojukwu teaching Hospital, Akwa, Anambra State, Nigeria, over a 6 month period. A structured questionnaire was administered to consenting patients to determine their sociodemographic variables and other characteristics. Visual acuity was determined with multiple optotype Snellen's chart at 6 metres in natural daylight. Using pen‐torch, the face, external eye examination, anterior segment examination and pupillary reaction to light were assessed. Magnified anterior segment examination was performed with slit‐lamp biomicroscope. All subjects underwent dilated fundoscopy (after instillation of tropicamide 0.5% eye drop) using Welch Allyn indirect ophthalmoscope.Results were analysed using SPSS version 22.0 Inc Chicago Illinois and represented in tables. Statistically significant P value was set at < 0.05

Results:

A total of 28 patients with age range of 6‐42 years were seen. 17(60.7%) were males, while 11(39.3%) were females. 8(28.6%) had at least one ocular anomaly comprising of refractory errors 3(10.8%), sickle cell retinopathy 4(14.3%) and glaucoma 2(7.1%). Only 2(7.1%) routinely visit eye clinic. SCD retinopathy was found to increase with age (P value = 0.005)

Summary/Conclusion:

SCD retinopathy was found more in the older age group despite the small sample size at a prevalence rate of 14.3%. Early initiation of routine ocular examination for SCD patients will be beneficial to avert these complications. More efforts should be made at educating the patients. Larger studies will be required to determine the actual prevalence of ocular anomaly in SCD patients in Anambra State.
PB2315 ECHO HEART AND TISSUE DOPPLER ASSESSMENT AS EARLY PREDICTORS OF CARDIAC INJURIES IN SICKLE CELL PATIENTS
Authors: Sayed N.
Published: June 2019

Background:

Major cardiac abnormalities in patients with sickle cell disease are related to volume overload effects of chronic anemia and ferritin level. Patients with SCA have reduced LV global longitudinal strain. In the detection of subclinical LV dysfunction in patients presenting with sickle cell disease, 2D speckle tracking and tissue Doppler echocardiography appears to be useful to assess systolic dysfunction and diastolic dysfunction respectively.

Aims:

The aim of our study was to evaluate cardiac performance in adult Egyptian asymptomatic sickle cell patients.

Methods:

The study was conducted on 30 SCD patients who were recruited from the hematology clinic of Kasr El‐Ainy Hospital‐ Cairo University and 15 healthy control who were age and sex matched. Two‐dimensional speckle tracking echocardioghraphy (STE), M‐mode, Doppler and tissue Doppler echocardiography (TDE) were are used to assess systolic and diastolic dysfunction in both groups. We used parameters (GLS, AP3L, AP2L, AP4L, EF, LVIDS and LVIDd) for systolic dysfunction. And for diastolic dysfunction we used parameters (medial and lateral E/e’, E/A ratio, Dt and S velocity).

Results:

our results revealed significant increase in LVIDS and LVIDd dimensions in cases compared to control group (P < 0.001). None had an ejection fraction below 55%. Diastolic dysfunction was elicited in 8 patients (26%). There was a statistically significant correlation between the serum ferritin level and lateral E/E’ ratio. There was a significant reduction in GLS (p value.026) and also apical segments longitudinal strain (p value.002) compared to control. There was statistically significant negative correlation between number of sickle cell crises per year and GLS (p = 0.040). There was a statistical significant inverse correlation between Global LV systolic function and hemoglobin level (p = 0.04).

Summary/Conclusion:

Cardiac performance should include assessment of both systolic and diastolic parameters as there could be alteration in LV diastolic filling indices even in absence of systolic dysfunction. Hence these indices can be used as an early marker for cardiac affection.

Speckle tracking echocardiography can recognize the cardiac involvement in really early stages and initiation of treatment and decreasing rate of painful crises to prevent irreversible myocardial dysfunction.
PB2318 DOUBLE HETEROZYGOSITY S/C AT HEMATOLOGY DEPARTMENT ANNABA HOSPITAL ‐ALGERIA
Authors: Djenouni A. , Grifi F.
Published: June 2019

Background:

Major sickle cell disorders account for 95% of the hemoglobinopathies supported at our service; double S/C heterozygosity is infrequent and represents only 6,4%.

Aims:

The objective of our work is to determine the prevalence and characteristics of acute and chronic complications that occurred in our patients during their follow‐up.

Methods:

This is a retrospective, descriptive study of 99 patients with double S / C heterozygosity and followed regularly since recruitment until December 2017.

The patients benefit from a regular follow‐up, every 04 months with an annual report aimed at detecting the chronic complications.

Results:

The average age of patients at the time of diagnosis is 21 years, with extremes of [02‐49 years].

The current average age of the cohort is 33 years, with extremes of [13‐66 years]

The majority of our patients come from Algerian‐Tunisian border area (79%).

During this monitoring, several variously associated complications were collected:

The acute complications are dominated by vaso‐occlusive crises in various locations, with exceptionnellent‐required hospitalization;

During follow‐up, 32.09% of our patients developed various chronic complications:

Summary/Conclusion:

Despite of an acute symptomatology low noise from the S/C, chronic complications are frequent, imposing the implementation of a multidisciplinary management strategy at our level.

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PB2359 THE ROLE OF AUTOLOGOUS STEM CELL TRANSPLANTATION IN INCREASING THE EFFICIENCY OF TREATMENT IN PATIENTS WITH MULTIPLE MYELOMA
Authors: Garifullin A. , Voloshin S. , Chubukina Z. , Kuzyaeva A. , Sсhmidt A. , Bessmeltsev S.
Published: June 2019

Background:

Multiple myeloma remains an incurable disease despite the emergence of new drugs. The search of optimal therapeutic strategy to increase life expectancy and remission in patients with this disease is continues. This is facilitated to the achievement of the maximum possible response and eradication of residual tumor load. Existing recommendations for the treatment of multiple myeloma approve the use of autologous stem cell transplantation (autoSCT) to improve disease control.

Aims:

To determine the impact of autologous stem cell transplantation on efficiency of treatment and tumor load in patients with multiple myeloma.

Methods:

The study included 89 patients with MM (median age 58 years, male/female – 1.12:1). The induction therapy with Bortezomib‐based regimens (VD, CVD, VMP, PAD) was used in 62/89 (69.6%) patients, Immunomodulator‐based regimens (Thal + D, RD, VRD, PomD) – in 22/89 (24.8%), chemotherapy – in 4/89 (5.6%). High dose therapy (Mel200) and autoSCT is carried out in 64/89 (71.9%) patients. The efficiency of therapy was evaluated according to IMWG criteria. Tumor load was determined by multicolor flow cytometry (5‐colors): 79 patients – after 4‐6 courses of induction therapy, 39 patients – after autoSCT; 29 patients – tumor load was performed in dynamics (before and after autoSCT).

Results:

The sex, age, the variant induction antimyeloma therapy did not affect on response rate and tumor load. CR was achieved in 28/89 (31.4%) before AutoSCT and 50/89 (56.2%) after AutoSCT (p > .05). However, high dose therapy (Mel200) and AutoSCT has increased the possibility of achieving complete response in dynamics: before AutoSCT in 5/29 (17.2%), after AutoSCT – in 13/29 (44.8%) (p<.05).

Tumor load after 4‐6 courses of induction therapy (n = 79) was 0.28 ± 0.34 for CR + sCR, 1.65 ± 2.66 for VGPR, 2.10 ± 2.53 for PR.Tumor load after HDT (Mel200) and AutoSCT (n = 39) was 0.06 ± 0.07 for CR + sCR, 0.15 ± 0.46 for VGPR, 0.17 ± 0.22 for PR.

The effect of autoSCT on PFS in 39 patients. The median PFS was 97 months in the group “tumor load after autoSCT <0.01%”, 70 months – in the group “tumor load without autoSCT <0.01%”, 50 months – in the group “tumor load after autoSCT >0.01%” and 25 months – in the group “tumor load without autoSCT >0.01%”(p = .006).

Examination of tumor load in the dynamics in 29 patients showed that autoSCT reduces the level of tumor load (before autoSCT = 2.27 ± 0.20, after autoSCT – 0.14 ± 0.01). Tumor load <0.01% after autoSCT was detected more often than before autoSCT (14/29 and 5/29 patients, respectively)(p<.05).

Summary/Conclusion:

AutoSCT increase the efficiency of therapy, reduce tumor load and increase the duration of progression free survival in patients with multiple myeloma.

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PB2369 THE METHOD OF STORAGE OF AUTOLOGOUS PERIPHERAL BLOOD HEMATOPOIETIC STEM CELLS
Authors: Rysev G. , Kuzyaeva A. , Voloshin S. , Shuvaev V. , Schmidt A. , Garifullin A. , Kuvshinov A. , Mikhaleva M. , Linnikov S.
Published: June 2019

Background:

High‐dose chemotherapy (HDCT) followed by autologous transplantation of hematopoietic stem cells (auto‐HSCT) is an important component of treatment in multiple myeloma (MM). There is a standard method of controlled cryopreservation of HSC suspension. We found that the storage of native HSC suspension with temperature fluctuations from +3 °C to +5 °C for 5 days does not significantly affect the content of CD34+ cells in the product, the index 7AAD– (7‐AAD (7‐aminoactinomycin – D) is a fluorescent marker that penetrates damaged cell membranes and binds to double‐stranded DNA. Through 7AAD does not penetrate intact membranes, so living cells are not stained 7AAD with flow cytometry), and colony‐forming ability (CFA) of HSC, as well as the recovery time of hematopoiesis in MM patients after auto‐HSCT.

Aims:

To evaluate the effectiveness and safety of the method of storage of non‐cryopreserved peripheral blood stem cells.

Methods:

26 patients with MM were included in this study(male/female ratio 1.36:1). All the patients get standard immunochemotherapy programs and were in remission at the time of auto‐HSCT. Patients were divided into two groups depending on the method of stem cell storage: group 1 – non‐cryopreserved (n = 10), group 2 – cryopreserved (standard) (n = 16). An effectivity and safety were evaluated in such parameters as the number of CD34+ and 7AAD‐ cells, CFA after apheresis and before reinfusion of HSC. Also we evaluated the number of platelet concentrate transfusions, the timing of engraftment of granulocytic and megakaryocytic blood sprouts, the length of hospital stay after auto‐HSCT, the number of complications.

Results:

The results are presented in the comparison table of evaluated parameters.

Summary/Conclusion:

Thus, the proposed method of storage of HSC is not inferior to the traditional method with cryopreservation on such parameters as CD34+, 7AAD–, CFA, the number of platelet concentrate transfusions, terms of hematopoiesis restoration, length of hospital stay after HSCT, the number of complications.

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PB2380 PROGNOSTIC EVALUATION OF MULTIPLE MYELOMA PATIENTS SUBMITTED TO UP‐FRONT AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION FROM A SINGLE BRAZILIAN CENTER
Authors: Vivas Y. , Hungria V. , Santos K. , Chaoubah A. , Bustamante‐Teixeira M. , Vivas L. , Costa L. , Neto A.
Published: June 2019

Background:

Although autologous hematopoietic stem cell transplantation (AHSCT) has been performed in the context of multiple myeloma since the end of the 90's, response to therapy and patient survival are not homogeneous between different patients.

Aims:

In order to understand the factors involved in therapy response degree and survival on AHSCT performed as first‐line therapy, we conducted a retrospective analysis of myeloma multiple patients from asingle Brazilian center.

Methods:

All patients treated with AHSCT between May 2004 and October 2017were evaluated, excluding patients with prior transplant therapies or insufficient records data.Variables related to the International Staging System (ISS), Durie‐Salmon staging, clinical presentation at diagnosis, regimen used for induction, number of treatment cycles, induction and transplant treatment response assessmentwere studied.Event free survival (EFS) and overall survival (OS) were calculated and associated to analyzed factors.

Results:

Altogether, 137 patients were included in the study, with an average age of 52 years, 59,1% of them were male and 86,9% nonblack. Fifty two patients (38%) died, 6 (4,4%) of these during AHSCT. OS was 63,8% and EFS was 25,1% in 5 years.OS univariate analysis, as confirmed by multivariate analysis, was smaller in patients with anemia at diagnosis (p = 0,006).EFS, submitted to multivariate analysis, was smaller in patients treated with more than one line of induction chemotherapy.A positive response to induction therapy was the main factor related to a very good partial response and complete response to AHSCT (p = 0,001).

Summary/Conclusion:

Notwithstanding the great gap in knowledge over the successful myeloma multiple AHSCT factors, our data is similar to the ones found in literature, with anemia as an important independent prognostic factor to OS.
PB2391 MYOCARDIAL CONTRACTILE FRACTION BY MAGNETIC RESONANCE AND RISK OF CARDIAC COMPLICATIONS IN THALASSEMIA MAJOR
Authors: Meloni A. , Pistoia L. , Rosso R. , Bitti P. P. , Caini M. , Carollo A. , Armari S. , Gerardi C. , Roberti M. G. , Fiorenza M. F. , Sarli R. , Renne S. , Positano V. , Pepe A.
Published: June 2019

Background:

The analysis of cine cardiovascular magnetic resonance (CMR) images allows the quantification of the myocardial contraction fraction (MCF), a volumetric analog of myocardial deforming, that is calculated by dividing left ventricular (LV) stroke volume by LV myocardial volume.

Aims:

We verified the association of this new marker with myocardial iron overload and cardiac events (heart failure, arrhythmias and pulmonary hypertension) in thalassemia major (TM) patients.

Methods:

We considered 1492 TM patients (771 females, 31.06 ± 8.95 yrs) enrolled in the MIOT network. Myocardial iron overload (MIO) was quantified by the multislice multiecho T2∗ technique and biventricular morphological and functional parameters by cine sequences.

Results:

MCF values were significantly lower in males (0.94 ± 0.22 vs 1.01 ± 0.22; P < 0.0001) and decreased with increasing age (R = −0.129 P < 0.0001).

Patients with MIO (global heart T2∗ < 20 ms; N = 400) showed significant lower MCF values than patients without MIO (0.91 ± 0.20 vs 1.00 ± 0.22; P < 0.0001).

The patient population was randomly divided in two groups of equal size (N = 746).

In the first group 99 patients had at least one cardiac complication and at receiver‐operating characteristic (ROC) curve analysis a MCF ≤ 0.87 was the best predictor of cardiac complications (area under the curve 0.659, 95% CI 0.64 to 0.69; sensitivity 60%, specificity 67%).

In order to validate the result, the obtained value was tested in the remaining 746 patients (group 2). One hundred and thirty‐five patients had a cardiac complication and patients with a MCF ≤ 0.87 were more likely to have cardiac complications (odds ratio – OR = 2.73, 95%CI = 1.86‐4.01; P < 0.0001), also adjusting for cardiac iron (see Figure).

Summary/Conclusion:

Contractile function is reduced in patients with MIO. A MCF ≤0.87 can help to identify patients with a significant higher risk of adverse cardiovascular events, independently by the presence of MIO.

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PB2399 BETA THALASSEMIA MINOR PATIENTS: NOT TIRED, BUT DEPRESSED AND ANXIOUS
Authors: Eren R. , Karışmaz A. , Aslan C. , Doğu M. H. , Altındal S. , Yokuş O. , Suyanı E.
Published: June 2019

Background:

The quality of life has been influenced unfavourably and the prevalance of either depression or anxiety has been increased in beta thalassemia major (BTM) and beta thalassemia intermedia (BTI) patients. Yet, the studies including the quality of life in beta thalassemia minor (BTm) subjects are limited and just the depression was demonstrated to be frequent in individuals with BTm, compared to the healthy population. Likewise, the data regarding the presence of fatique and anxiety in adult BTm subjects is insufficient

Aims:

We aimed to investigate whether the severity of fatigue and the incidences of depression and anxiety of patients with BTm are different than healthy individuals using Fatigue Severity Scale (FSS) and Hospital Anxiety and Depression Scale (HADS).

Methods:

Seventy‐seven BTm subjects were included in this study. The individuals (n = 38) using any medications, alcohol or tobacco; and having any chronic diseases and sleeping disturbances, were excluded. The remaining 39 BTm subjects were enrolled in the study. They had also normal biochemical parameters, thyroid function tests and C‐reactive protein (CRP). Twenty‐five healthy control subjects who were matched with age, sex, marital status, educational status and body mass index (BMI), were also included.

Results:

The BTm and the control groups were comparable in terms of gender, age, BMI, educational status and marital status (p = 0.368, 0.755, 0.851, 0.785, 0.709 respectively) (Table 1). Fatigue Severity Scale score was ≥4 in 23 (59.0%) BTm subjects and in 15 (60%) control subjects (p = 1.0). HADS anxiety score was ≥10 in 20 (51.3%) BTm subjects and in 5 (20.0%) control subjects (p = 0.018) and HADS depression score was ≥ 7 in 20 (51.3%) BTm subjects and 6 (24.0%) healthy control subjects (p = 0.039). There was no correlation of hemoglobin with FSS score (p = 0.526, r = ‐0.105), HADS anxiety score (p = 0.703, r = ‐0.063) or HADS depression score (p = 0.718, r = ‐0.06) in BTm group (Table 2).

Summary/Conclusion:

We found that both depression and anxiety were higher in BTm patients than healthy individuals, but this difference was not feasible for fatigue.

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