EHA Library - The official digital education library of European Hematology Association (EHA)

Abstract

Abstract: EP817

Type: e-Poster

Background
Patients with myelodysplastic syndromes (MDS) require individualized treatment approaches, integrating the perception of patients. Thus, patient-reported outcomes (PROs) have been introduced, which include any information that comes directly from the patient such as symptom severity or quality-of-life (QoL). So far, core PROs have not been defined in MDS.

Aims
We aim (1) to identify QoL measurement instruments (QoL-MI) relevant in MDS, as a comprehensive source of potential core PROs, (2) to nominate core PROs for MDS by patients and hematologists.

Methods
We performed a comprehensive systematic literature search (PubMed, Cochrane Library, Scopus, Web of Science) to identify QoL-MI. Studies were screened by two independent reviewers and the observed QoL-MI were summarized in an evidence table. The domains and items within each of the observed QoL-MI were extracted and categorized into potential PROs by experienced researchers and clinical experts. To nominate core PRO, a two-round Delphi survey was performed among MDS patients from the outpatient clinics of Saint Louis Hospital in Paris and physicians from 18 different countries as part of the MDS-RIGHT project and the EUMDS Registry. Each outcome was ranked for importance, using a nine-point agreement scale and predefined criteria according to the 1-3/7-9 scoring. Missing values were imputed by the items’ median ranking and new outcomes suggested by at least two participants were included in the next round. We used Spearman's rank correlation coefficient (ρ) to determine the correlation of the mean PRO rankings between groups.

Results
The literature search resulted in 2,863 studies; 81 studies were included in our review. Overall, we identified twelve generic, six cancer-specific and two MDS-specific QoL-MI. The most frequently used instruments were EORTC QLQ-C30 (26 studies), FACIT-An (17), SF-36 (16), QoL-E (10), and FACIT-BMT (7). These five instruments composed 68% of the total 112 QoL-MI applications, whereas the MDS-specific QoL-MIs composed only 10%. Forty potential core PROs were extracted from the QoL-MI and applied in the two-round Delphi survey. In the patients’ Delphi survey, we obtained 40 responses in the first round followed by 38 in the second round. Results showed an agreement for inclusion of “general QoL” into the core PROs set. Further outcomes ranked highly include “fatigue“, “general health“, “confidence in health care services“, “weakness“, and “disease knowledge“. Among the physicians, we obtained 38 responses in the first round and 32 in the second Delphi survey. Three PROs fulfilled the inclusion criteria: “general QoL”, “transfusion dependence”, and “ability to work/activities of daily living”. The Spearman coefficients showed moderate correlation between mean physician and patient scores (1st round: ρ=0.51, p<0.001; 2nd round: ρ=0.54, p<0.001). Lower agreement was observed for “disease knowledge” and “confidence in health care services”, both ranked notably higher by the patients.

Conclusion
QoL-MI, particularly MDS-specific QoL-MI are underused in current MDS practice. This first consensus set of core PROs selected by patients and hematologists is useful to promote quality of care in daily practice and forms the basis for patient-centered care.

Session topic: 10. Myelodysplastic syndromes - Clinical

Keyword(s): MDS, Outcome measurement

Abstract: EP817

Type: e-Poster

Background
Patients with myelodysplastic syndromes (MDS) require individualized treatment approaches, integrating the perception of patients. Thus, patient-reported outcomes (PROs) have been introduced, which include any information that comes directly from the patient such as symptom severity or quality-of-life (QoL). So far, core PROs have not been defined in MDS.

Aims
We aim (1) to identify QoL measurement instruments (QoL-MI) relevant in MDS, as a comprehensive source of potential core PROs, (2) to nominate core PROs for MDS by patients and hematologists.

Methods
We performed a comprehensive systematic literature search (PubMed, Cochrane Library, Scopus, Web of Science) to identify QoL-MI. Studies were screened by two independent reviewers and the observed QoL-MI were summarized in an evidence table. The domains and items within each of the observed QoL-MI were extracted and categorized into potential PROs by experienced researchers and clinical experts. To nominate core PRO, a two-round Delphi survey was performed among MDS patients from the outpatient clinics of Saint Louis Hospital in Paris and physicians from 18 different countries as part of the MDS-RIGHT project and the EUMDS Registry. Each outcome was ranked for importance, using a nine-point agreement scale and predefined criteria according to the 1-3/7-9 scoring. Missing values were imputed by the items’ median ranking and new outcomes suggested by at least two participants were included in the next round. We used Spearman's rank correlation coefficient (ρ) to determine the correlation of the mean PRO rankings between groups.

Results
The literature search resulted in 2,863 studies; 81 studies were included in our review. Overall, we identified twelve generic, six cancer-specific and two MDS-specific QoL-MI. The most frequently used instruments were EORTC QLQ-C30 (26 studies), FACIT-An (17), SF-36 (16), QoL-E (10), and FACIT-BMT (7). These five instruments composed 68% of the total 112 QoL-MI applications, whereas the MDS-specific QoL-MIs composed only 10%. Forty potential core PROs were extracted from the QoL-MI and applied in the two-round Delphi survey. In the patients’ Delphi survey, we obtained 40 responses in the first round followed by 38 in the second round. Results showed an agreement for inclusion of “general QoL” into the core PROs set. Further outcomes ranked highly include “fatigue“, “general health“, “confidence in health care services“, “weakness“, and “disease knowledge“. Among the physicians, we obtained 38 responses in the first round and 32 in the second Delphi survey. Three PROs fulfilled the inclusion criteria: “general QoL”, “transfusion dependence”, and “ability to work/activities of daily living”. The Spearman coefficients showed moderate correlation between mean physician and patient scores (1st round: ρ=0.51, p<0.001; 2nd round: ρ=0.54, p<0.001). Lower agreement was observed for “disease knowledge” and “confidence in health care services”, both ranked notably higher by the patients.

Conclusion
QoL-MI, particularly MDS-specific QoL-MI are underused in current MDS practice. This first consensus set of core PROs selected by patients and hematologists is useful to promote quality of care in daily practice and forms the basis for patient-centered care.

Session topic: 10. Myelodysplastic syndromes - Clinical

Keyword(s): MDS, Outcome measurement

By clicking “Accept Terms & all Cookies” or by continuing to browse, you agree to the storing of third-party cookies on your device to enhance your user experience and agree to the user terms and conditions of this learning management system (LMS).

Cookie Settings
Accept Terms & all Cookies