Haematological malignancy: Are we measuring what is important to patients? A systematic review of quality‐of‐life instruments

Abstract

The wide range of health‐related quality‐of‐life (HRQoL) instruments used in haematology makes it challenging for haematologists and other care team members in practice to select, use and understand the scoring system and finally interpret the results. The main objectives of this study were to: (a) provide a comprehensive list of quality‐of‐life issues important to patients suffering from haematological malignancies, identified through the literature; (b) provide a list of health‐related quality‐of‐life (HRQoL) instruments used in haematological malignancies in both daily clinical practice and research; and (c) evaluate the relevance and comprehensibility of the identified instruments in haematological malignancies. Systematic literature review of two databases, followed by addition of articles by manual searching, was carried out. The articles focusing on the primary studies, which have used semi‐structured/structured interviews or surveys to identify issues important to HM patients, and other studies describing the results of testing measurement properties, such as reliability, validity and responsiveness of the instruments currently used to evaluate the HRQoL in different HMs, were included. Fifty‐seven studies reported development and validation of 30 HRQoL instruments, which have been used in haematology. Twenty‐four studies were identified using qualitative methods to report HRQoL issues and symptoms from a patient's perspective. No identified instrument captured all the issues identified from the qualitative studies. None of the instruments reviewed appeared to have been developed for use in clinical practice and specifically for patients with HM, except MyPOS. Furthermore, measurement properties were established, largely, in clinical trial scenarios. There is a need for development of a new HRQoL instrument entirely based on involvement of patients with haematological malignancies.

Development of a Novel Hematological Malignancy Specific Patient-Reported Outcome Measure (HM-PRO): Content Validity

“…Several studies have previously reported that HM has a high impact on patients' HRQoL (Persson et al, 2001;Holzner et al, 2004;Santos et al, 2006;Mols et al, 2007;Shanafelt et al, 2007;Strasser-Weippl and Ludwig, 2008;Johnsen et al, 2009). A recent systematic review reported that there are 30 HRQoL instruments currently used in hematology and none of these instruments captures all the issues important to these patients (Goswami et al, 2019). Furthermore, this review also reports that a barrier to using patient-reported outcomes (PRO) in clinical practice is the diversity of such instruments.…”

“…follicular lymphoma patients (Osborne et al, 2015;Davies et al, 2017), and therefore cannot be applied to other HM. Among the identified 30 HRQoL instruments, only partial evidence on the content validity was identified for EORTC QLQ-MY24, FACT-Leu, EORTC Leu, FACT-Lym, QoL-E (Goswami et al, 2019). Up to 18% of the patients resported missing items for EORTC QLQ-MY24 (Goswami et al, 2019).…”

Introducing patient‐reported outcome in the acute leukemia quality registries in Sweden

“…These are also the most frequently used in hematological research. 17,18 Still, intervention studies are sparse, and the use of PRO to improve treatment of hematological malignancies has not been well established. [19][20][21][22][23] In 2011-2012, the Blood Cancer Registry 24 decided to start a pilot project using PRO in the acute myeloid leukemia (AML) and acute lymphoblastic leukemia (ALL) registries.…”

Haematologists’ experiences implementing patient reported outcome measures (PROMs) in an outpatient clinic: a qualitative study for applied practice

“…The framework builds on the assumption that the patient-doctor relationship is crucial in providing holistic, patient-centred care and alleviating the symptom burden caused by the disease or treatment. PROMs can help identify symptom burden and inform the decision-making process, leading to initiation of supportive care [1][2][3].…”

“…Adaption of PROMs has also been linked to measurement uncertainties, including content validity (content relevance and content coverage) and the psychometric properties of the PROM(s) [17,18]; inadequate measurement properties could potentially lead to clinicians using invalid outcomes when consulting with patients, which again could potentially be harmful and detrimental to the patient-doctor relationship. Important knowledge gaps remain regarding the complexity of PROMs usage and how to adapt them across different settings for routine clinical care [3,4]. Despite an extensive volume of literature on the use of PROMs for routine clinical care, it is difficult to reach firm conclusions due to the broad variety of interventions within settingspecific studies [4,19,20].…”