Research Group: Registry Studies and Clinical Trials II
Research Group Lead
- Dr. rer. biol. hum. Michael Lauseker, Dipl.-Stat.
- Kirsi Manz, Dipl. Phys., M.Sc.
- Dr. rer. biol. hum. Kathrin Halfter, Dipl.-Biol.
- Arthur Gil, M.P.H.
- Denise Kohn
- Elvis Nkwetta
- Annett Schmitt, Dipl. Biochemistnach oben
The group is responsible for overseeing the biostatistics of four currently recruiting clinical trials on Chronic Myeloid Leukemia (CML- the TIGER, BODO, DasaHIT, and ENDURE studies), one study on multiple myeloma (GERMAIN study), and one study on the secondary prevention of type 1 diabetes (Fr1da Study).
In addition to the above-mentioned clinical trials, data is being collected and analyzed for three registry studies on CML. The registry studies are the NAUT study, the EUTOS Registry, and the EURO-SKI study.
Data management using electronic case report forms (eCRFs)
The team began programming eCRFs in 2012 and eCRFs have been implemented in four studies – TIGER, GERMAIN, BODO, and NAUT Study.
Methods, Planning, and Analysis of Registry Studies and Clinical Trials
As part of the biostatistical support, the research team is responsible for the statistical sections of study protocols. High quality standards are placed on sample size estimation and adjustment methods for tests with multiple hypotheses, as well as the development of analysis methods to ensure a solid methodological basis beyond standard procedures.
A major aim of many projects is, and was, the analysis of potential prognostic factors for various treatment outcomes. The EUTOS registry, for example, provided large data sets of CML patients treated with Imatinib, which led to the development of two widely recognized and valid prognosis systems: The EUTOS score, used to predict the disease remission rate at 18 months(https://www.leukemia-net.org/content/leukemias/cml/eutos_score/index_eng.html) and the ELTS score for the long-term survival probabilities (https://www.leukemia-net.org/content/leukemias/cml/elts_score/index_eng.html). In addition, a prognostic score for AML patients has been proposed (https://www.leukemia-net.org/content/leukemias/aml/aml_prt_score/index_eng.html).
Survival and Competing risk analysis
The primary endpoints of most studies in leukemia research are time-dependent variables. For this reason our research team closely monitors the developments of new methods for the survival analysis of overall survival time, specifically those that account for adjustments for competing risks. As a result our group has authored several publications on the Cox-Cure model, the Fine-Gray model, the modeling of multiple stages, the analysis of left truncated data, and relative survival probabilities.
Health Services Research
Using routine medical data, our group is also studying how this acquired knowledge is implemented into the routine clinical treatment of patients with CML or myelodysplastic syndrome (MDS).
Currently ongoing studies Projects, launched since 2012
- CML V TIGER Study: A prospective, randomized, open-labeled, multicenter clinical trial on the Treatment optimization of newly diagnosed Ph/BCR-ABL positive patients with chronic myeloid leukemia (CML) in chronic phase with nilotinib vs. nilotinib plus interferon alpha induction and nilotinib or interferon alpha maintenance therapy.
- Germain Study: A prospective, randomized, double-blinded, multicentered clinical trial comparing Lenalidomid maintenance therapy vs placebo in elderly patients with multiple myeloma
- Bosutinib Dose-finding study (Bodo Study): Multicenter, open-label single arm phase II study testing the tolerability and the efficacy of bosutinib step-in dosing in chronic phase CML patients intolerant or refractory to previous imatinib, nilotinib or dasatinib therapy
- NAUT Study: Multicenter prospective trial after first unsuccessful treatment discontinuation in chronic myeloid leukemia (CML) estimating the efficacy of nilotinib in inducing the persistence of molecular remission after stopping TKI a 2nd time
- DasaHIT study: A prospective, randomized, open-labeled, dose-finding study to investigate the tolerability and efficacy to give a medication 5 out of 7 days vs. giving the medication daily.
- Endure Study: Efficacy and safety of pegylated proline interferon alpha 2b (AOP2014) in maintaining deep molecular remissions in patients with chronic myeloid leukemia (CML) who discontinue ABL-kinase inhibitor therapy - a randomized phase II, multicenter trial with post-study follow-up
- Prognostic research using the European Treatment and Outcome Study (EUTOS) registry – a registry for patients treated with imatinib
- Prognostic research to obtain molecular remission in CML patients after treatment discontinuation (EURO-SKI study)
- Fr1Da Study: A Prospective, randomized study to investigate the oral administration of insulin as a secondary prevention for patients with type 1 diabetes
- Projects to study the reality of medical care for CML patients in Bavaria using data from public health insurance companies
- Projects to study the distribution of new MDS diagnoses in Bavaria using data from public health insurance companies