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Quality of Life Assessment After HSCT for Pediatric and Adults

  • Anna Barata
  • Heather Jim
Open Access
Chapter

Abstract

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.

34.1 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.

34.2 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.

34.3 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.

34.3.1 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

(a) General

European Quality of Life- 5 Dimensions (EQ-5D-5L) (van Reenen and Jansen 2015)

Aim

Health status

Items

6

Domains/subscales

Mobility, self-care, usual activities, pain, anxiety, depression

Results

Profile of each of the domains assessed, and an index of the health status. Higher scores indicate better health status

Translations

Available in more than 130 languages

Medical Outcomes Study-Short Form (MOS SF-36) (Ware et al. 1994)

Aim

QoL

Items

36; shorter versions feature 12 items (SF-12) or 8 items (SF-8)

Domains/subscales

General health, physical, role, emotional and social functioning, mental health, pain, vitality

Results

Physical Component Score; Mental Component Score and Global Score. Higher scores indicate better QoL

Translations

Available in more than 170 languages

Patient-Reported Outcomes Measurement Information System (PROMIS) (Cella et al. 2010)

Aim

Mental, physical, and social health and QoL in healthy populations as well as those with chronic conditions

Items

Multi-item measures varying in length and complexity; for example, PROMIS-29 has 29 items, PROMIS-43 has 43 items, PROMIS-57 has 57 items

Domains/subscales

Each subscale measures a single domain; PROMIS Profile measures assess multiple domain

Results

Higher scores indicate more of the concept being measured. Measures use standardized T-score metric against normative data for the US population

Translations

Available in Spanish and several other languages

(b) Cancer and HCT specific

European Organization for Research and Treatment of Cancer QoL Questionnaire Core 30 (EORTC QLQ-C30) version 3.0 (Aaronson et al. 1993)

Aim

QoL in cancer

Items

30 items

Domains/subscales

Functional scales, symptom scale and a QoL scale

Results

Higher scores in functional and QoL scales indicate better wellbeing. Higher scores in the symptom scale indicate worse symptomatology

Translations

Available in more than 100 languages

Functional Assessment of Cancer Therapy—Bone Marrow Transplant (FACT-BMT) (McQuellon et al. 1997)

Aim

QoL in HCT

Items

47

Domains/subscales

Consists of the FACT-G (Cella et al. 1993) and the BMT concerns subscale

Results

Higher scores indicate better QoL

Translations

Available in more than 38 languages

Functional Assessment of Cancer Therapy—General Scale (FACT-G) (Cella et al. 1993)

Aim

QoL in cancer

Items

33

Domains/subscales

Physical, functional, social and emotional well-being

Results

Higher scores indicate better wellbeing and global QoL

Translations

Available in more than 60 languages

34.3.2 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

(a) General

Child Health Questionnaire (CHQ) (Landgraf et al. 1996)

Aim

QoL

Versions

Parent-reported versions feature 50 items (CHQ-PF50) or 28 items (CHQ-PF28) and are intended for parents of children aged 5–18 years. The child-report version (CHQ-87) has 87 items and is appropriate for children aged 10–18

Domains/subscales

Global health, physical functioning, role/social-physical functioning, bodily pain/discomfort, role/social-emotional functioning, role/social -behavior, parental impact -time, parental impact -emotional, self-esteem, mental health, global behavior, family activities, family cohesion, and changes in health

Results

Higher scores indicate higher physical and psychosocial wellbeing

Translations

The CHQ-PF50 and CHQ-PF28 are available in more than 80 languages, and the CHQ-87 to 34

Patient-Reported Outcomes Measurement Information System (PROMIS) (Hinds et al. 2013)

Aim

Health and QoL in healthy populations as well as those with chronic conditions

Versions

Multi-item measures varying in length and complexity: PROMIS-25 has 25 items, PROMIS-37 37 items, and PROMIS-49 49 items. PROMIS measures are child- and parent-reported. Child-report measures are intended for children aged 8–17, and parent-report for children 5–17

Domains/subscales

Physical, mental and social health, and a global QoL score

Results

Higher scores indicate more of the concept being measured. PROMIS use standardized T-score metric against normative data for the US population

Translations

Children and proxy measures are available in Spanish and in several other languages

Pediatric Quality of Life Inventory (PedsQL™) 4.0 Generic Score Scales (Varni et al. 2001)

Aim

QoL in healthy children or those diagnosed with an acute or chronic disease

Versions

Parent-report form for children aged 2–4 has 21 items, and child and parent reports for children aged 5–18 have 23 items

Domains/subscales

Physical, emotional, social, and school functioning

Results

Physical health summary score; Psychosocial health summary score; Total score. Higher scores indicate better QoL

Translations

Available in more than 70 languages

(b) Cancer and HCT specific

Child Health Rating Inventories (CHRIs)-and Disease-Specific Impairment Inventory-Hematopoietic Stem Cell Transplantation (DSII-HCT) (Parsons 2005)

Aim

The disease specific (DSII-HCT) module assesses QoL of childhood HCT survivors

Versions

10-item module intended to child-report (aged 5–12), adolescent-report (13–18) and parents-report (5–18)

Domains/subscales

Items are grouped in three domains reported by parents and patients to be most salient to the HCT experience: worry, hassless, and body image

Results

Higher scores indicate better QoL

Translations

The questionnaire is available in English

Peds Quality of Life Cancer Module 3.0 (PedsQL CM™) (Varni et al. 2002)

Aim

QoL in children with cancer

Versions

Parent-report form for children aged 2–4 has 25 items, child and parent reports for children aged 5–7 has 26 items, and child and parents reports for children more than 8 years has 27 items

Domains/subscales

Pain and hurt, nausea, procedural anxiety, treatment anxiety, worry, cognitive problems, perceived physical appearance and communication

Results

Higher scores indicate better QoL

Translations

Available in more than 100 languages

The Behavioral, Affective and Somatic Experiences Scales (BASES) (Phipps et al. 1994)

Aim

QoL during the acute phase of HCT

Versions

There are separate versions to be completed by nurses (BASES-N), parents (BASES-P) and children (BASES-C). The BASES-N and BASES-P have 38 items and the BASES-C has 14 items. The questionnaire is intended to be used in child aged 5–17

Domains/subscales

Somatic distress, mood disturbance, compliance, quality of interactions and activities

Results

Higher scores indicate more distress/impairment

Translations

Available in English

34.4 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.

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Authors and Affiliations

  1. 1.Department of HematologyHospital de la Santa Creu i Sant Pau, Universitat Autònoma de BarcelonaBarcelonaSpain
  2. 2.José Carreras Leukemia Research InstituteBarcelonaSpain
  3. 3.Department of Health Outcomes and BehaviorMoffitt Cancer CenterTampaUSA

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