12th Congress of the European Hematology ... - Haematologica

12 th Congress of the European Hematology Association
0601
EUROPEAN LEUKEMIANET - INTEGRATION OF THE LEADING NATIONAL LEUKEMIA
NETWORKS (CML, AML, ALL, CLL, MDS, CMPD) AND THEIR INTERDISCIPLINARY
PARTNER GROUPS IN EUROPE
S. Saussele, 1 K. Adam, 1 M. Baccarani, 2 M.C. Béné, 3 Th. Buechner, 4
A. Burnett, 5 G. Finazzi, 6 C. Fonatsch, 7 E. Gluckman, 8 N. Goekbuget, 9
D.J. Grimwade, 10 T. Haferlach, 11 M. Hallek, 12 J. Hasford, 13
A. Hochhaus, 1 D. Hoelzer, 9 P. Ljungman, 14 U. Mansmann, 13
D. Niederwieser, 15 S. Serve, 4 B. Simonsson, 16 T. J. M. De Witte, 17
R. Hehlmann1 1 III. Medizinische Klinik Mannheim, MANNHEIM, Germany; 2 Università di
Bologna, BOLOGNA, Italy; 3 Faculté de Médecine, NANCY, France; 4 Universitätsklinikum
Münster, MÜNSTER, Germany; 5 University Hospital of Wales,
CARDIFF, United Kingdom; 6 Ospedali Riuniti di Bergamo, BERGAMO, Italy;
7 Medizinische Universität Wien, WIEN, Austria; 8 BMT and Eurocord/Netcord
Registry, PARIS, France; 9 II. Medizinische Klinik Frankfurt, FRANKFURT,
Germany; 10 King's College London, LONDON, United Kingdom; 11 MLL
Münchner Leukämielabor GmbH, MÜNCHEN, Germany; 12 1. Medizinische
Klinik, Universität Köln, KÖLN, Germany; 13 IBE Universität München,
MÜNCHEN, Germany; 14 Karolinska University Hospital, STOCKHOLM,
Sweden; 15 Universität Leipzig, LEIPZIG, Germany; 16 Univerity Hospital, UPP-
SALA, Sweden; 17 University Medical Center Radboud, NIJMEGEN, Netherlands
Leukemias serve as a model for a variety of diseases and possess
exemplary relevance for basic research and patient care. Leukemia
research and therapy have achieved high standards and even a leading
position in several European countries. A true European world leadership,
however, had not been accomplished due to national fragmentation
of leukemia trial groups, diagnostic approaches and research activities
and the lack of central information and communication structures.
To overcome national fragmentation the European LeukemiaNet (ELN)
was started in 2002. It is funded within the 6th EU-Framework Program
since 2004. The ELN now integrates 92 leading national leukemia trial
groups (CML, AML, ALL, CLL, MDS and CMPD), 86 interdisciplinary
partner groups (diagnostics, treatment research, registry, guidelines) and
industry partners across Europe to form a cooperative network for
advancements in leukemia-related research and health care. The trial
groups and their partners which represent several thousand participating
centers and ten thousands of study patients treated within the trial
groups, form the backbone of the network. The network with its integration
and interdisciplinary cooperation brings together 133 participating
institutions and approximately 1000 researchers from 24 countries.
The network consists of 16 thematically distinct workpackages. Of
these, six deal with the various disease entities and represent subnetworks
on their own. Seven workpackages represent interdisciplinary
platforms, which provide the support and research expertise required for
high quality networking and excellence. Three central service workpackages
provide central communication, information and management
services for the whole network and support integration. Scientific highlights
of accomplished work include: 1. Establishment of information,
communication and management structures. Communication is mostly
accomplished via the information center (ELIC) and by the network
management center (NMC) through annual symposia, regular networkand
WP-meetings, an ELN website, and biannual newsletters. A European
Leukemia Trial Registry (ELTR) was developed in accordance with
the guidelines of the International Committee of Medical Journal Editors
and the WHO. ELTR will be connected to the WHO Meta-Registry,
as soon as the WHO has defined definitive interfaces for data-transfer.
2. European registries have been started for CML, ALL, ET and MDS. 3.
Clinical trials on an European level are ongoing. 4. Quality control
rounds and consensus recommendations in diagnostics on a European
level were achieved e.g. for molecular monitoring in CML, cytogenetic
analysis in CLL and morphological diagnosis of leukemias. 5. Several
guidelines and management recommendations were completed and in
part published, e.g. management recommendations for CML, recommendations
for harmonizing methodology for detecting BCR-ABL transcripts
and kinase domain mutations, guidelines for microarray analyses,
guidelines on definition of transplant-associated microangiopathy
(TAM), recommendations for standardizing indications for SCT, guidelines
on prophylaxis and empirical antifungal therapy in neutropenic
leukemia patients. The main goals for the first three years have been
achieved and the ELN is well prepared for further integration advances
in research, diagnosis and treatment of leukemia according to its goals.
224 | haematologica/the hematology journal | 2007; 92(s1)
0602
PRIMARY PROPHYLAXIS WITH PEGFILGRASTIM WAS MORE COST-EFFECTIVE THAN
FILGRASTIM IN PATIENTS WITH NON-HODGKINS LYMPHOMA RECEIVING CHOP-21 IN
THE UK
R. Pettengell, 1 P. Booth, 2 J. Malin, 3 C. Atchison, 4 Z. Liu, 5 Q. Doan, 5
A. Lalla, 5 R. Dubois5 1 St George's Hospital Medical School, LONDON, United Kingdom; 2 Amgen
Ltd, CAMBRIDGE, United Kingdom; 3 Amgen Inc., THOUSAND OAKS,
USA; 4 Amgen Europe GmbH, ZUG, Switzerland; 5Cerner LifeSciences, BEV-
ERLY HILLS, USA;
Background. In the 2006 ASCO and EORTC guidelines, primary prophylaxis
with granulocyte colony-stimulating factors (G-CSFs) is recommended
when the overall risk of febrile neutropenia (FN) from chemotherapy
and patient-related factors is equal to or greater than 20%. Without G-CSF,
FN incidence among patients receiving CHOP-21 chemotherapy is 17-
50%. Although the daily first-generation G-CSF filgrastim and the longacting
second-generation G-CSF pegfilgrastim are both commonly used,
in practice filgrastim is often administered for shorter-than-recommended
courses, which has been shown to be associated with less clinical benefit.
Aims. We evaluated the cost-effectiveness of pegfilgrastim vs. filgrastim
used for 11 days (as used in the randomised trials demonstrating efficacy)
and 6 days (as often used in clinical practice) in patients with aggressive
NHL receiving CHOP-21 in the UK. Methods. A decision-analytic
model was constructed from the National Health Service’s perspective.
The study time horizon was life-time. Model inputs, including FN risk
(varied by days of filgrastim use), FN case-fatality, relative dose intensity
(RDI), impact of RDI on survival, and utility scores were from a comprehensive
literature review and expert panel validation. Costs were from
official price lists or literature and included drugs, drug administration,
FN-related hospitalisations, and subsequent medical costs. NHL mortality
data were from literature; all-cause mortality data were from official statistics.
The model simulated three clinical scenarios: scenario 1 included
the impact of prophylaxis with pegfilgrastim or filgrastim on FN risk; scenario
2 included the impact of a difference in FN risk on FN-related mortality;
scenario 3 included a differential impact on RDI and long-term survival.
Using data from a meta-analysis (pegfilgrastim vs. 11 days of filgrastim)
and observational studies (pegfilgrastim vs. 6 days of filgrastim), we
estimated the absolute risk of FN in patients receiving pegfilgrastim
decreased by 6.5 percentage points (19.6 vs. 13.1%) vs. 11-day filgrastim,
and by 12 percentage points (25.1 vs. 13.1%) vs. 6-day filgrastim. Model
robustness was tested using sensitivity analyses. Outcomes were measured
as incremental cost-effectiveness ratio (ICER) including £ per percentage
(absolute) FN risk decreased, £ per FN event avoided, £ per life-year
gained (LYG), and £ per quality-adjusted life-year (QALY) saved. Results.
Pegfilgrastim was cost saving compared with 11-day filgrastim in all scenarios.
Compared with 6-day filgrastim, the ICER was £6,675 per FN
avoided or £ 67 per 1% decrease in absolute risk of FN in scenario 1. The
ICER was £ 29,438/QALY saved in scenario 2. In scenario 3, when all
potential benefits of G-CSF were considered, the ICER became £
7,699/QALY saved (Table 1).
Table 1. Cost-effectiveness of pegfilgrastim vs. 6-day filgrastim.
Results were sensitive to the relative risk of FN for 6-day filgrastim vs.
pegfilgrastim and study time horizon. Summary and conclusions. In the UK,
pegfilgrastim was cost saving compared with 11-day filgrastim use. The
cost of pegfilgrastim vs. 6-day filgrastim at £7,699-£29,438/QALY gained
was favourable compared with the £30,000/QALY threshold commonly
used in the UK. With primary prophylaxis against FN in NHL patients