The challenges of outcome research

Cognitive pharmacy trials seek to identify interventions that benefit patients. The potential benefits of an intervention are primarily evaluated by outcome measures. The question then is: What is the optimal outcome measure? Unfortunately, the question remains unsolved. Several factors must be taken into consideration when conducting outcome research—particularly within cognitive pharmacy trials. The interventions are often complex and non-specific, and seek to improve symptom control, optimise the use of medications and reduce medication-related risks. “Hard” endpoints, such as mortality and hospital admissions, may not be the optimal outcome measures, since cognitive pharmacy interventions are unlikely to result in changes in these measures. Instead, adverse drug events or “soft” endpoints, such as quality of life, drug-related problems and patient satisfaction may be appropriate choices of outcome measures. Finally, it is not only outcome measures that may pose a challenge when conducting outcome research; other essential components include study design, type of intervention, the patient population, etc.     

Treatment of elderly and very old patients with AML

Acute Myeloid Leukemia (AML) is a genetically heterogeneous malignant disease mainly occurring in elderly patients. The prognosis of patients over 60 years of age, which represent the majority of patients, which are diagnosed, remains poor. The main reasons for the poor outcome are the in-creasing number of comorbidities, organ impairments and nevertheless the unfavorable genetic features, pre-existing myelodysplastic changes and rising multi-drug resistance. It is also more likely that older and frail patients are not included into clinical trials and treatment decisions have to be made on general assumptions.     

New approaches in graft versus host disease (GvHD) management

Graft versus host disease (GVHD) remains the major cause of morbidity and nonrelapse mortality (NRM) after allogeneic hematopoietic stem cell transplantation (HSCT). Although the understanding of its biological basis is continuously improving and candidate target pathways therefore increasingly emerge, severe, steroid-refractory GVHD remains an unsolved issue with a rather poor prognosis in clinical practice thus far. With the exception of the Janus kinase (JAK) 1/2 inhibitor, ruxolitinib, which has demonstrated considerable effectiveness in steroid-refractory GVHD in a multicenter survey, few advances have been recognized in this field during recent years. Therefore, developments toward better, more individualized GVHD prophylaxis are needed. This, however, requires a more precise knowledge of risk factors for severe GVHD. One such emerging risk factor is the homozygosity for HLA-C group 1 killer cell immunoglobulin-like receptor (KIR) ligands. In addition, ongoing investigation into the predictive value of immunogenetic polymorphisms and post-transplant biomarkers will contribute to individually designed and adaptable GVHD management in the future, hopefully relying mainly on prophylactic and pre-emptive measures.