1 Introduction

Methodological advances in the HCT field have increased the population of survivors worldwide. However, HCT is associated with significant morbidity that impairs survivors’ recovery and adversely affects their QoL. A significant body of literature has addressed QoL after HCT and highlights significant deficiencies in physical, psychological, social, and role functioning both in adult and pediatric survivors (Pidala et al. 2010). These data are clinically relevant as they help to understand the impact of HCT on patient’s lives. Clinically, assessment of QoL can inform patient education and be used to evaluate the benefit of supportive care interventions.

2 QoL Assessment

QoL can be considered a patient-reported outcome (PRO). PROs are defined by the US Food and Drug Administration (FDA) as the “measurement of any aspect of a patient’s health status that comes directly from the patient, without the interpretation of the patient’s response by a clinician or anyone else” (US Food and Drug Administration 2009). Thus, PROs specifically describe the impact that HCT has on patients’ lives and provide information unavailable from other sources (Kurosawa et al. 2017; Russell et al. 2006). PROs are also used in pediatric populations, although parents or other proxies might be used as source of information when children are unable to report their own QoL. However, the use of patients’ own reports is clearly recommended because significant discrepancies are found when comparing patients’ self-reported QoL to reports of physicians, parents, or other proxies (Kurosawa et al. 2017; Russell et al. 2006). In general, measures to assess patient- and proxy-reported QoL are questionnaires.

These instruments can be broadly categorized as general or disease- or procedure-specific. General measures assess QoL of the general population and can also be administered to specific populations, such as HCT recipients. These questionnaires allow comparisons of QoL across populations, such as between HCT survivors and individuals without cancer. In contrast, disease- and procedure-specific instruments examine specific aspects of the health conditions assessed. These measures capture specific PROs that are likely to be important to patients.

3 Measures to Assess QoL in Adults and Pediatric Patients Undergoing HCT

There are numerous measures assessing QoL on adults and pediatric HCT recipients. Measures used have been both general and disease-specific. The following sections list some of the most common used questionnaires in the field of HCT.

3.1 Adults

Interest in assessing QoL in adult HCT recipients is reflected in the variety of measures used to assess this outcome. However, there is a need for the scientific community to reach consensus about which questionnaires to use in order to facilitate comparison across studies (Shaw et al. 2016). Table 34.1 summarizes alphabetically some of the most common questionnaires to assess QoL in adults.

Table 34.1 QoL questionnaires assessing QoL in adult HCT survivors
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3.2 Pediatrics

There is less research on QoL on pediatric patients than adult patients. Initial pediatric studies focused on a single aspect of functioning, such as psychosocial and physical limitations. It was not until the early 1990s that pediatric QoL began to be addressed as a multidimensional construct. Most of the measures used in pediatric studies were originally developed to be used in the general population or in children with specific illnesses. Table 34.2 lists alphabetically the most common measures used to assess QoL in pediatric population.

Table 34.2 QoL questionnaires assessing QoL in pediatric HCT survivors
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4 Challenges when implementing QoL assessment

Improvement in patients’ QoL is included among the strategic goals of major cancer organizations such as the American Society of Clinical Oncology and regulatory agencies such as the FDA and the European Medicines Agency. Recognition of the importance of the patient experience is reflected in the increasing incorporation of patient-reported QoL measures in observational research and clinical trials. However, some aspects should be considered when implementing patient-reported QoL measures.

Historically, studies and clinical trials performed in the USA have often used the FACT instruments, whereas studies performed in Europe have chosen the EORTC. This divergence makes results difficult to compare (Shaw et al. 2016), although efforts are underway to map common QoL measures such as the EORTC QLQ-C30 and FACT-G to one another (Young et al. 2015). Second, the mode of administration should also be considered. PRO measures have traditionally been administered by paper and pencil, but new technologies offer the potential to use electronic measures. Electronic measures administered before or during a clinic visit allow results to be available at the time of consultation and may facilitate symptom monitoring to guide supportive treatment. One example is the PROMIS instrument, which is available using computer adaptive testing or through REDCap software. Computer adaptive testing selects questions based on the previous responses that patients have provided to approximate the construct being measured in the fewest number of questions. The implementation of routine assessment of patients’ QoL on clinical care and clinical trials has the potential to improve patients’ well-being.

Key Points

  • Assessing HCT survivors’ QoL is essential in order to know the impact that the HCT, its morbidity, its treatments, and related interventions have on survivors’ well-being.

  • Enhanced efforts should be made to in order to include QoL assessment in routine clinical practice. Engaging clinicians in using QoL assessments, potentially by means of electronic administration, as well as broadening the interpretation of their scores into the clinical field, might facilitate incorporation.

  • Further efforts should elucidate to what extent QoL results are incorporated into management decisions, treatment recommendations, and patients’ education.

  • Additional efforts should also be made to include QoL outcomes in clinical trials.

  • The incorporation of QoL assessment into clinical and research practice has the potential to improve HCT outcomes.