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10th EHA Congress
Third Meeting of the EHA
Working Group on
Friday, 3 June 2005
18:00 – 20:00
Stockholm International Fairs
10th EHA Congress
Third Meeting of the EHA Working Group on
Friday, 3 June 2005
18:00 – 20:00
Stockholm International Fairs
Title of the presentation
- Introduction and presentation of the meeting P. Imbach (Switzerland) - Immunopathology and immunotherapy in ITP - Diagnostic approach to platelet function disorders - The European Survey on Evans Syndrome: an T. Kühne (Switzerland) - ICIS-PARC Study: update and new proposals corticosteroid treatment in previously untreated ITP patients: a proposal for randomised, multicentric Members of the WG at - Proposals and brief communications Diagnostic approach to platelet function disorders

Marco Cattaneo. Unità di Ematologia e Trombosi, Ospedale San Paolo. Università di
The diagnostic approach to disorders of platelet function is based on a two-step strategy. The first step include simple, rapid and cost-effective tests, which are sensitive to the most common platelet function disorders. Since light transmission aggregometry is not sufficiently sensitive to defects of platelet secretion, which are the most common disorders of primary haemostasis, platelet function should be screened by lumiaggregometry. Additional tests include inspection of the peripheral blood smear and clot retraction. The combined analysis of the results of these tests wil al ow to raise a diagnostic hypothesis, which wil then be verified in the second step of the diagnostic strategy, including specific confirmatory tests. Due to the many pre-analytical and analytical variables that affect the results of platelet functions tests, both steps of the diagnostic strategy for platelet disorders should be done in specialized centres. The European survey on Evans Syndrome: an update

Marc Michel M. Department of Internal Medicine, Centre Hospitalier Universitaire Henri

Evans syndrome (ES) is a rare disorder defined by the combination of autoimmune
hemolytic anemia (AIHA) and immune thrombocytopenic purpura (ITP), both conditions
occurring either simultaneously or sequential y. To better describe the main features of this
syndrome, we have previously reviewed 21 cases of ES seen at our institution over a 15
year-period. AHAI was defined by a haemoglobin level (Hb) 10g/dL, with features of
hemolysis and a positive direct antiglobulin test (DAT). ITP was defined according to the
usual criteria set for ASH and a platelet count (plt ct) < 100 x 109/L. Al patients had to
have a normal marrow aspirate and the delay between AIHA and ITP could not exceed 10
years. Sixteen women (76%) and 5 men (mean age: 53 ± 23.5 years) were included. ITP
and AHAI occurred simultaneously in 15/21 cases (71%), ITP preceded the onset of AIHA
in 5 cases (mean delay between both conditions = 3.4 years), whereas AHAI occurred
before ITP in a single case. The mean Hb level at AHAI diagnosis was 6 g/dL and the
mean plt ct at AITP diagnosis was 27x109/L. None of the patients have had a life-
threatening haemorrhage whereas AHAI lead to a cardiac complication, including 2 cases
of myocardial infarction (MI), in 6 of the 11 patients (55%) aged of 60 or above. Overal ,
ES was considered as being “idiopathic” in 11/21 cases (53%), systemic lupus
erythematosus (SLE) was diagnosed in 6 patients (aged from 15 to 43 years), a “lupus-like
syndrome” (3 ARA classification criteria) was found in 3 and one patient had an IgA
deficiency. After a median fol ow-up of 3.5 years, a B-cel Non-Hodgkin Lymphoma (NHL)
occurred in 2 patients and a myelodysplastic syndrome in 2. Al patients but one received
corticosteroids as a first-line therapy, patients with an underlying SLE were given
hydroxychloroquine in combination. For the management of AIHA, danazol was given to 5
patients (2 cases of hepatitis), 5 patients received immunosuppressive drugs, 1 patient
achieved a complete remission (CR) with rituximab, and 4 underwent splenectomy (1 CR).
At the time of analysis, 4 patients were in treatment-free CR, 9 patients were in CR on
treatment, 1 patient was in partial remission and 7 patients (33%), al aged of 60 or above
had died. The causes of death were: NHL (2), MDS (2), MI (1), septic shock on day 17
post-splenectomy (1) and oesophagus cancer (1). In conclusion, in our experience, ES is
mainly associated with SLE in young adults and can precede the onset of NHL or MDS in
elderly patients. While AITP is seldom severe, AIHA may lead to life-threatening complications especial y in elderly patients. However, since this was a single retrospective study, recruitment bias cannot be excluded. In order to better characterize the main features and the outcome of ES in adults, a European survey is currently underway with the support of the Working Group on thrombocytopenias. More than 50 cases have been analysed and preliminary data wil be presented at the meeting.
ICIS-PARC Study: update and new proposals
Thomas Kühne, MD, University Children’s Hospital Basel, Switzerland.
Idiopathic or immune thrombocytopenic purpura (ITP) is a bleeding disorder occurring in
both children and adults. Although the pathogenesis of the disease is now better
understood, ITP remains a diagnosis of exclusion. The remaining disorder, cal ed “ITP”, is
a col ection of various thrombocytopenias causing disorders with a heterogeneous
background. The etiological and pathophysiological uncertainties of ITP together with a
significant lack of data regarding management of the disease, which was revealed and
systematical y analysed when preparing practice guidelines by the American Society of
Hematology (George et al., 1996), let a group of hematologists establish the
Intercontinental Childhood ITP Study Group (ICIS) in 1997.
ICIS ( provides a network of physicians from al continents who
are involved in the research and management of patients with ITP. The aims of the group
are to promote research by wel designed prospective trials, information and
communication, and to provide a platform for physicians involved in the management of
patients with ITP. ICIS started with a project cal ed Registry I, representing a prospective
international data col ection of children with newly diagnosed ITP. Registry I was closed
when almost 3,000 children from 147 institutions of 39 countries were registered, with a
total of 220 participating physicians. Registry II represents an attempt to qualify and
quantify bleeding with the hypothesis that the phenotype of ITP is measurable and
represents a clinical and research endpoint superior to the platelet count. Registry II
systematical y analyses the frequency, location, timing and severity of major bleeding in
children with newly diagnosed ITP. Registry I is now closed, and patient accrual for
Registry II is completed. Splenectomy Registry is stil open for patient registration. The
main objectives include the evaluation of the appropriate timing of the procedure, pre-
operative preparation of the patients, perioperative management issues, such as
technique of splenectomy, and postoperative aspects, such as the evaluation of
complications, and short- and long-term evaluation of platelet values.
The Pediatric and Adult Registry on Chronic ITP (PARC-ITP) was activated by ICIS 2004
and is open for registration for children and adults with ITP. This online Registry
( represents a database serving as the main part of the study
with the potential to add subsequent side-studies as modules, which wil build the “trees in
the park”. The aims of the PARC-ITP study include the analysis of the heterogeneity of ITP
and the search of new selection criteria for future studies. Secondary aims are the study of
the natural history of ITP and the validation of the diagnosis. The PARC-ITP study is a
prospective international multicenter registry. The data col ection wil be managed by the
central data office of ICIS in Basel, Switzerland.
A study of the genetic aspects of ITP represents the first side-study to PARC-ITP. It wil be
added as an amendment to the PARC-ITP protocol. This study aims to identify new
genomic regions predisposing to or influencing the course of ITP in children and adults.
For this purpose from each patient a blood sample (3-5 ml EDTA blood) wil be stored
frozen and DNA wil then be analyzed by different laboratories in USA, UK, and
Switzerland. The data of patients with ITP wil be compared with a cohort of controls of the
same size. The study protocol from PARC-ITP can be downloaded from The amended text of the protocol wil be available soon, as it
has been submitted to the local ethical committee.
Rituximab in first-line ITP therapy
Nichola Cooper (University Col ege Hospital, NHS Trust, London, United Kingdom) Rituximab therapy is increasingly being used in the treatment of immune thrombocytopenia (ITP). A number of studies have suggested between 30 and 60% response rate with as many as 30% of patients having a complete and lasting remission (CR). It has a number of advantages over other therapies. For example, unlike steroids, it does not appear to have serious adverse side effects. Secondly, unlike IVIG and IV anti-D, it can induce a CR for > 1 year from infusion (with some patients stil maintaining a normal platelet count more than 5 years from treatment). However, the exact place of rituximab in patients with ITP remains unclear. Should it be used upfront or reserved for those not responsive to splenectomy? This partial y depends on the outcome of patients with ITP, the pathogenesis of ITP and the long-term consequences of rituximab therapy. There is very little data on patients with ITP treated upfront with rituximab. Occasional case reports show it has activity in patients with acute ITP not responsive to first line therapy such as steroids and IVIG. However, there are no control ed, or uncontrol ed trials. While most of the initial trials using rituximab as a single agent in non-malignant conditions suggests it is safe, with few adverse side effects, there have been increasing reports of unusual infections in patients treated for haematologic malignancies when rituximab is combined with chemotherapy. Occasional case reports have also described unusual chest infections in patients treated for ITP. This should be considered when contemplating B cel depletion therapy. Furthermore, at least 30% of adults with ITP are expected to go into complete remission within the first 6 months without rituximab, and as a significant proportion of patients may not have a plasma derived anti-platelet factor and are likely to have a purely T cel mediated disease, this may not be of any value. In summary, there is stil not enough data to recommend rituximab therapy as initial therapy in patients with ITP. It does however show promise in patients who are refractory to traditional first line therapy such as steroids and immunoglobulin. There is also increasing evidence suggesting its safety as a presplenectomy agent. Further randomized control ed trials are required. High dose dexamethasone vs standard corticosteroid
treatment in previously untreated adult ITP patients: a GIMEMA
randomized, multicenter study
Francesco Rodeghiero, Department of Hematology, San Bortolo Hospital, Vicenza, Italy- On behalf of the Writing Committee (M. G. Mazzucconi, F. Rodeghiero, F. Mandel i et al)
The best initial treatment of adult patients with immune thrombocytopenic purpura (ITP) is
stil not supported by prospective control ed trials. Oral corticosteroids (1-2 mg/Kg b.w./day
for 2 - 4 weeks) represent the “standard” time-honored treatment, but some studies
suggest that intravenous immune globulin (IVIG) and pulsed high-dose steroids may be
more effective, for short term and/or long term response, even though no direct
comparison has been made. Recently, Cheng and Col eagues (NEJM, 2003) published
their not-control ed experience with high-dose dexamethasone (HDD), given in a single 4-
day therapy course (40 mg/day, oral y) in previously untreated adult patients with ITP,
showing very encouraging results (85% initial response; 42% sustained response at 6
months of fol ow-up). A not-yet published GIMEMA (Italian Group on Adult Hematology
Diseases) pilot study confirms these results (Mazzucconi, personal communication), with a
response after 60 days in 81 of 95 (89%) ITP patients (around 50% children) treated with
at least 3 cycles of HDD (at 14 days intervals).
Given these data, GIMEMA ITP study group has decided to start a prospective,
randomized trial of first-line therapy in previously untreated adult patients comparing oral
prednisone, 1 mg/kg b.w/day for 4 weeks to HDD (40 mg/day for 4 days oral y for 3
cycles), in order to evaluate the rate of sustained response. Patients relapsing or not-
responding after standard therapy wil be switched to HDD. Patients relapsing or not
responding after HDD wil be treated with anti CD 20 antibodies (375 mg/m2 once a week
for 4 weeks), to evaluate the effectiveness of this drug as a splenectomy-sparing strategy.
This second part of the study is facultative. The study is due to start next Autumn and it is
open to the suggestions and to the participation of the European Centers through the EHA
– WG on Thrombocytopenias (contact F. Rodeghiero at:


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