title: Systematic assessment of decision-analytic models for chronic myeloid leukemia.
abstract: BACKGROUND
  Several tyrosine kinase inhibitors (TKIs) are approved for the treatment of chronic myeloid leukemia (CML). Decision-analytic modeling can help to extrapolate data from short-term clinical trials and also consider quality of life when evaluating different treatment strategies.
  
  
  OBJECTIVE
  Our goal was to describe and analyze the structural and methodological approaches of published decision-analytic models for various treatment strategies in CML and to derive recommendations for the development of future CML models.
  
  
  METHODS
  We performed a systematic literature search in electronic databases (MEDLINE/PreMEDLINE, EconLit, EMBASE, NHS EED, and Tuft's CEA Registry) to identify published studies evaluating CML treatment strategies using mathematical models. The search was updated in August 2013.
  
  
  METHODS
  The models were required to compare different treatment strategies in relation to relevant clinical and patient-relevant health outcomes [e.g., life-years gained, quality-adjusted life-years] over a defined time horizon and population.
  
  
  METHODS
  We used standardized forms for data extraction, description of study design, methodological framework, and data sources for each model.
  
  
  RESULTS
  We identified 18 different decision-analytic modeling studies. Of these, 17 included economic evaluations. Modeling approaches included decision trees, Markov cohort models, state-transition models with individual (Monte Carlo) simulations, and mathematical equations. Analytic time horizons ranged from 2 years to a lifetime. Treatment strategies compared included bone marrow or stem cell transplantation, conventional chemotherapy, interferon-α, and TKIs. Only one model evaluated a second-generation TKI. Most models did not report a model validation. All models conducted deterministic sensitivity analyses and four reported a probabilistic sensitivity analysis.
  
  
  CONCLUSIONS
  Articles that were not published in English or German were not included in this review. Our literature search was restricted to published full-text articles in certain databases. Therefore, publications that met our inclusion criteria but were published in different databases, different languages, or as abstracts only may have been missed.
  
  
  CONCLUSIONS
  While several well-designed models of CML treatment strategies exist, there remains a need for the assessment of the long-term efficacy and cost effectiveness of novel treatment options such as second-generation TKIs. Additionally, these models should be validated using independent data.
authors.affiliation: Division of Public Health Decision Modelling, Health Technology Assessment and Health Economics, ONCOTYROL-Center for Personalized Cancer Medicine, Innsbruck, Austria, ursula.rochau@umit.at.
authors.forename: Ursula
authors.initials: U
authors.lastname: Rochau
authors.forename: Ruth
authors.initials: R
authors.lastname: Schwarzer
authors.forename: Beate
authors.initials: B
authors.lastname: Jahn
authors.forename: Gaby
authors.initials: G
authors.lastname: Sroczynski
authors.forename: Martina
authors.initials: M
authors.lastname: Kluibenschaedl
authors.forename: Dominik
authors.initials: D
authors.lastname: Wolf
authors.forename: Jerald
authors.initials: J
authors.lastname: Radich
authors.forename: Diana
authors.initials: D
authors.lastname: Brixner
authors.forename: Guenther
authors.initials: G
authors.lastname: Gastl
authors.forename: Uwe
authors.initials: U
authors.lastname: Siebert
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000970:Antineoplastic Agents / Q000191:economics / Q000627:therapeutic use; D016026:Bone Marrow Transplantation / Q000191:economics; D003661:Decision Support Techniques; D005260:Female; D006801:Humans; D016898:Interferon-alpha / Q000191:economics / Q000627:therapeutic use; D015464:Leukemia, Myelogenous, Chronic, BCR-ABL Positive / Q000191:economics / Q000628:therapy*; D008297:Male; D008390:Markov Chains; D008875:Middle Aged; D018803:Models, Economic; D047428:Protein Kinase Inhibitors / Q000191:economics / Q000627:therapeutic use*; D011505:Protein-Tyrosine Kinases / Q000037:antagonists & inhibitors*; D011788:Quality of Life; D019057:Quality-Adjusted Life Years; D033581:Stem Cell Transplantation / Q000191:economics; D016896:Treatment Outcome
chemical_list: D000970:Antineoplastic Agents; D016898:Interferon-alpha; D047428:Protein Kinase Inhibitors; D011505:Protein-Tyrosine Kinases