Table 3 Effectiveness outcomes
Outcome variable(s) | Measure(s) and data collection procedures | Data source and reporter (when applicable) |
|---|---|---|
Primary Outcome | ||
 Re-hospitalisation rate | Number of events after the initial post-alloSCT discharge per patient in the first year post-alloSCT | EHR |
Secondary Outcomes | ||
 Healthcare utilization costs | (1) Calculated from the payers’ perspective based on standardised unit costs of resources in Switzerland [84, 85, 91] | EHR |
(2) Medical records and an adapted version of the generic FIMA© self-reporting questionnaire for elderly persons [83]: 7 items, asking retrospectively for number of visits to physicians, days/hours of ambulatory healthcare visits, days/hours of home care services received, days/hours of support by family caregivers, type(s) and duration(s) of rehabilitation therapy, reason(s) and duration(s) of inpatient days (including intensive care unit stays and/or emergency room visits), and current type of insurance. | Paper survey (pat.) | |
 Length re-hospitalizations | (1) The total length of inpatient re-hospitalizations in the first year after alloSCT is the total number of hospitalized days (planned and unplanned) after initial post-alloSCT discharge until end of study, like reported in all medical reports and the generic FIMA© self-reporting questionnaire for elderly persons [83] | Paper survey (pat.) and EHR |
 Medication adherence (implementation & persistence dimension [67]) | (2) Implementation & persistence dimension [67] will be assessed using the Basel Assessment of Adherence to Immunosuppressive Medication Scale (BAASIS©): a validated self-report measure assessing adherence to implementation issues (e.g., drug holidays; 4 items; yes/no) and persistence/discontinuation (1 item; yes/no) [87]. | Paper survey (pat.) |
(3) Daily intake (date and time) of immunosuppressive medication will be monitored electronically via the MEMS® Button, [86] which has been indicated previously as the preferred electronic monitoring device [63]. Patients will receive the button shortly before discharge and are instructed on how to use it at home until their immunosuppressants are discontinued. Data will be password-protected and stored on the MEMS® Adherence Software database, which provides an overview of the electronically compiled dosing history per patient [86]. | MEMS® Button [86] | |
 Treatment burden | German version of the PETS© self-reporting questionnaire [88]: nine multi-item domain scales, each measuring the burden of one aspect of chronic illness treatment on a 4- or 5-point Likert-type response scales regarding a 4-week recall time frame: medical information (7 items); medications (7 items); medical appointments (3 items); health monitoring (2 items); interpersonal challenges (4 items); health care expenses (5 items); difficulty with health care services (7 items); role/social activity limitations due to self-management (6 items); and physical/mental exhaustion due to self-management (5 items). Raw domain scores are transformed to a standardised 0-to-100 metric, with higher scores indicating greater treatment burden. | Paper survey (pat.) |
 Health-related Quality of Life | Measured using the EQ-5D-5L© [89], covering five dimensions (mobility, self-care, usual activities, pain/discomfort and anxiety/depression) and including the EQ–Visual Analogue Scale (VAS), on which individuals rate their overall perceived health state (scale of 0 to 100) | Paper survey (pat.) |
 Quality-Adjusted Life Year (QALY) | Calculate QALY: Generating these requires the HRQL preference weight (obtained from the EQ-5D-5L© value set for Germany EQ-VT v. 2.0) [92] and time in days between HRQL measurements [93]. QALY scores range from 1 (perfect health) to 0 (dead) [94]. | Paper survey (pat.) |
 GvHD | Incidence and grade of chronic and acute GvHD | EHR |
 Overall survival rate | Overall survival rate from start of study participation | EHR |
 Conditioning regimen | Treatments used to prepare a patient for stem cell transplantation (e.g., chemotherapy, monoclonal antibody therapy, and radiation to the entire body) | EHR |
 Donor match/type | Human leukocyte antigen (HLA) tissue type | EHR |
 Disease | Primary disease | EHR |
 If death | Date and cause of death | EHR |
 If relapse | Date of relapse | EHR |