Myelodysplastic syndromes (MDS) form a heterogeneous group of malignant haematological disorders of the elderly with a median age at diagnosis over 70 years. The life expectancy in western countries has risen to more than 80 years and cancers are stepping ahead as the leading cause of death. Due to an increasingly aging population and higher diagnostic accuracy, incidence and prevalence of MDS are continuously rising with growing impact on healthcare systems. Consequently, the appropriate use of health care resources and the assessment of their impact on relevant outcomes are of increasing interest.
Several evidence-based guidelines and recommendations (G&Rs) have been published for adult MDS patients. However, publishing G&Rs does not necessarily translate into better “quality” of care. There is a growing interest in understanding reasons and the consequences of guideline-adherence/non-adherence as retrospective studies are reporting deviation form published G&Rs. However, a systematic, prospective investigation in this field has not been done so far. This is mainly due to insufficient definitions, standardization and measurability of “good quality” of care, which is hampering further developments. Moreover, the currently available datasets from national cancer registries are not suitable for detailed health service research analyses as they are frequently incomplete and limited by many sources of biases. To this aim, the Swiss MDS Study Group (SMSG) implemented the Swiss MDS Registry/Biobank (SMRB) platform aiming to provide a standardized, systematic and shared infrastructure for data and samples collection to foster national and international collaborative projects.
The SMSG initiated the observational I-CARE for MDS study sponsored by Swiss Cancer Research and the Swiss Group of Clinical Cancer Research (SAKK). The study is conducted by the SAKK on the shared SMRB platform with the objectives to 1) define relevant guideline-based indicators (GBIs) as measurable elements of practice performance for appropriate care, 2) assess the level of adherence and reasons for non-adherence to GBIs and 3) measure the impact of adherence/non-adherence to GBIs on relevant outcomes for MDS patients in an observational, prospective study (Figure 1).
We are currently finalizing the development of GBIs (first objective) in collaboration with the European Leukemia Net MDS working group and other internationally acknowledged experts in the field. We systematically screened G&Rs from cooperative MDS groups as well as cancer care accreditation/certification programs (i.e. NCCN, DGHO, ELN, EBMT, NMDSG, GFM, NICE and others). All relevant information was extracted by a structured procedure and summarized as candidate GBIs in a handbook (including basic and additional information as well as definitions of numerator, denominator, source of data and time-points for assessment). We applied a RAND technique with a two-step DELPHI rating procedure to find an expert consensus for the clinically most relevant GBIs (REF: Coulter J et al, J Health Serv Res Policy, 2018). The expert panel group (EPGs) members consisted of 17 internationally acknowledged MDS experts, 7 additional health-professionals (3 nurses, 1 pharmacologist, 1 physiotherapist, 1 psychologist and 1 epidemiologist) and 3 patient advocates. Candidate GBIs were rated using a 9-point Likert-like scale for Relevance, Understandability, Measurability, Behavioral orientation and Attainability (RUMBA-criteria). They were ranked according to the agreement score, defined as % of all EPG members scoring all RUMBA-criteria in the first tertile (9, 8, or 7). The final set of GBIs for the management of adult MDS patients, covering the domains of diagnostics, treatment as well as provider characteristics, will be soon publically available.
This set of GBIs was developed in the context of the I-CARE for MDS project and represents the most comprehensive integration of current practice based G&Rs. The selection of these most relevant GBIs was based on a structured consensus process involving internationally acknowledged experts. These GBIs shall be further tested for applicability/operability, level and reasons for adherence/non-adherence as well as the potential impact of GBI adherence/non-adherence on relevant outcomes in retro- and prospective MDS cohorts, such as the EU-MDS. Combined with a core outcome set, GBIs will eventually enable to systematically monitor, compare and optimize appropriateness of health care provided to MDS patients in everyday clinical practice. As such, they will be important for the systematic and standardized assessment of practice performance for appropriate care in MDS patients and could be used as counterbalancing “value” or “quality” in economically driven health care systems with limiting resources.