Abstract

Chronic monthly transfusions are a lifesaving preventative therapy for many patients with sickle cell disease; however, the burden of this therapy for patients and families is high. In the United States, there is overlap in the population affected by sickle cell disease and those with the greatest burden of social needs. Hematology providers caring for patients with SCD have an opportunity to screen for and mitigate social determinants of health, especially in those receiving chronic transfusion therapy given the frequent interactions with the healthcare system and increased demand on already potentially limited resources. Given the complexity of the treatment and medication regimens, providers caring for patients receiving chronic transfusions should implement universal strategies to minimize the impact of low health literacy, as this therapy imposes a significant demand on the health literacy skills of a family. Despite the social and literacy burden of this intervention, it is reassuring that quality of life is preserved as patients with SCD on chronic transfusion therapy often report higher health related quality of life than their peers receiving other disease modifying therapies.

Learning Objectives

  • Understand the impact of social determinants of health on a chronically transfused population with sickle cell disease, as well as available screening tools.

  • Recognize the role of health literacy in complex medical decision-making and universal communication strategies to improve comprehension in patients of all health literacy levels.

  • Identify health-related quality of life measures that are affected by chronic transfusion therapy.

Clinical case

The patient is a 2 year-old girl with hemoglobin SS. She has been followed in the hematology clinic since birth. She was born at home at an estimated 32 weeks to a 27-year-old G5P5 mother who had limited prenatal care. Her older siblings are all half-siblings and range from 2 to 12 years old. There are no other siblings with sickle cell disease (SCD). The patient has had 1 admission for acute chest syndrome and several admissions for fever and dactylitis. Her mother is unemployed and spends her time caring for her family at home. Her father is not involved in her care. The patient has Medicaid and is enrolled in the Special Supplemental Nutrition Program for Women, Infants, and Children. Her mother expressed concern about lead exposure at her 9-month hematology visit, given peeling paint in their home, but she subsequently failed to routinely follow up. Social workers connected with the patient’s mother, who identified transportation as a significant barrier to attending clinic visits. The family received parking vouchers and gas cards but continued to miss appointments because of difficulty finding care for the other children in the home. Unfortunately, no extended family members were available to alleviate that burden, because many worked at the time when clinic was open. After she failed to come for her first transcranial Doppler, the medical team contacted child protective services with concerns about medical neglect.

Social determinants of health

Many facets of patient health are influenced by factors outside of the hospital or clinic setting. Health is heavily shaped by our families, homes, neighborhoods, and communities. These external factors are referred to as social determinants of health (SDHs), and they affect how patients interact with the health care system. In the United States, SDHs have been prioritized as part of the national health agenda in Healthy People 2020, where the goal is to “create social and physical environments that promote good health for all.”1  The World Health Organization has also prioritized policies and prevention strategies that affect SDHs because 23% of deaths globally can be attributed to factors in the environment.2 

SDHs are very broad and often overlap, but they can be organized into general categories. Healthy People 2020 divides the elements of SDHs into 5 domains: education, social and community context, health and health care, neighborhood and built environment, and economic stability.1  SDHs such as financial stress, food insecurity, and housing instability have all been linked to poor health outcomes and early mortality,3,4  and families often have resource needs across multiple domains.5  Adversity experienced in the home may be categorized as a separate domain, because adverse childhood experiences (ACEs) have been shown to independently shape health outcomes later in life.6  ACEs include exposure to child abuse, childhood neglect, and household dysfunction. They have been linked to health risk behaviors in a variety of cultural and economic settings.7 

Poverty, or low socioeconomic position, underlies many social problems and increases the risk of experiencing ACEs.8  In the United States, where structural inequity and systemic disenfranchisement have led to generational cycles of poverty that disproportionately affect racial and ethnic minorities, patients affected by SCD overlap with those at greatest risk for resource needs.9  In a quality improvement study that implemented universal screening for SDHs in a US pediatric hematology clinic, 156 screens were completed, and 66% were positive for at least one unmet need. On average, families reported 1.2 unmet needs (range 0 to 5), with food insecurity, difficulty paying for utilities, and a desire for more education being the most common.9  Our patient case highlights some of the domains affected at baseline by our patients and families, which may be further taxed upon starting chronic transfusion therapy given the intensity and frequency of therapy. Caregivers lose more days from work, have higher transportation costs, have additional medication costs, and have a high demand for childcare if other siblings cannot come to clinic visits. For pediatric patients, absences from school affect their educational attainment and interactions with peers.

Socioeconomic disparities, such as lack of transportation and inadequate health insurance, have been shown to affect access to comprehensive care for SCD. These factors prevent patients from receiving appropriate and timely screening for the adverse effects of SCD, increasing the medical burden and health care costs of this disease.10  In a post hoc analysis of baseline data from the Silent Cerebral Infarction Transfusion trial, every $10,000 increase in reported household income was associated with 5% fewer annualized emergency room (ER) visits.11  In a national sample of pediatric patients with SCD in the United States, children who visited the ER in the previous year were more likely to live in a household with a single mother; however, other SDHs included in the analysis were not significant predictors of ER use.12  In that study, authors postulate that the generally high burden of social disadvantage among children with SCD may explain their results.

A number of available screening tools for SDHs query elements from a variety of domains. See Table 1 for additional information about SDH screening tools for pediatric patients and families. Implementation of such screening tools can quickly identify the patients and families at highest risk. However, it is unclear which SDH or SDH domain has the greatest influence on patient health outcomes and health care utilization. Also, the critical SDH domain probably varies by country and location, given the differing availability of resources. For example, health care access is more critical in the United States, where universal health care does not exist.

Table 1.

Examples of screening tools for SDHs

Screening toolAdministration method, time to completeSummary of items evaluatedAdditional notesAge range (y)References
Safe Environment for Every Kid Parent Screening Questionnaire Paper and pencil or computer, 3-4 min, 16 or 20-item questionnaire with yes/no options Food insufficiency Part of a larger intervention that includes training of clinicians, identification of community resources, and social work support. 0-18 13,14  
Smoke alarm needed 
Poison control contact needed 
Parental stress and depression 
Parental intimate partner violence 
Parental drug or alcohol problems 
Tobacco use in home 
Help with the child is needed 
HealthBegins Upstream Risks Screening Tool Face-to-face interview, 6 min Food insufficiency  NR 15  
Difficulty making ends meet or basic needs 
Parental employment 
Parental education 
Concerns about the child’s learning or behavior 
Parental physical activity and consumption of nutritious foods 
Housing and neighborhood concerns 
Religious or organizational affiliation 
Parental social support 
Parental marital status 
Health Related Social Problems Screener Computer or tablet, 20 min Food insufficiency Survey used branching logic and ranged from 99 to 166 questions based on responses provided. Referrals were automatically generated. This format was highly acceptable to most families. 0-6 16  
Housing instability 
Parental employment 
Household income 
Insurance status 
Problems receiving health care 
Intimate partner violence 
Housing conditions 
iScreen Computer, face-to-face, or phone interviews, 10 min, 23 items assessed via Likert scale Economic instability Informants using the computer-based platform disclosed more needs, both in quantity and as a higher degree of endorsement than those performed face-to-face. 0-18 17,18  
Concerns about lack of child care or services 
Physical or mental health coverage 
Concerns about tobacco smoke or physical activity 
Concerns about housing or transportation 
Immigration status 
Drug or alcohol problems in the home 
Family member incarceration 
Violence toward the child in the home 
Addressing Social Key Questions for Health Tool Paper and pencil, 4-5 min, 13-question screen Food insufficiency Addresses ACEs and unmet social needs but low capture rate of ACEs. 0-18 19  
Housing insecurity 
Need for legal aid 
Parental education and employment 
Child witnessed violence and bullying 
Child physical and sexual abuse 
Child separation from caregiver 
Parental mental illness or substance abuse 
WE CARE Survey Instrument Paper and pencil, 4-5 min, 6 questions Food insufficiency  0-10 20,21  
Housing instability 
Parental education and employment 
Difficulty paying bills 
Lack of child care 
Family Needs Screening Program Paper and pencil, 1-page questionnaire with mostly yes/no options Housing instability Available in multiple languages targeting recent immigrants. Resource navigators supported families following a positive screen. Electronic referrals to community-based services also provided. 0-18 22  
Difficulty with paying bills, meeting basic needs, or getting public benefits 
Need for legal aid 
Parental education 
Help with child or elder care 
Health literacy 
Experiences of discrimination 
Available social supports 
Parental alcohol, drug, and tobacco use 
Housing conditions 
Screening toolAdministration method, time to completeSummary of items evaluatedAdditional notesAge range (y)References
Safe Environment for Every Kid Parent Screening Questionnaire Paper and pencil or computer, 3-4 min, 16 or 20-item questionnaire with yes/no options Food insufficiency Part of a larger intervention that includes training of clinicians, identification of community resources, and social work support. 0-18 13,14  
Smoke alarm needed 
Poison control contact needed 
Parental stress and depression 
Parental intimate partner violence 
Parental drug or alcohol problems 
Tobacco use in home 
Help with the child is needed 
HealthBegins Upstream Risks Screening Tool Face-to-face interview, 6 min Food insufficiency  NR 15  
Difficulty making ends meet or basic needs 
Parental employment 
Parental education 
Concerns about the child’s learning or behavior 
Parental physical activity and consumption of nutritious foods 
Housing and neighborhood concerns 
Religious or organizational affiliation 
Parental social support 
Parental marital status 
Health Related Social Problems Screener Computer or tablet, 20 min Food insufficiency Survey used branching logic and ranged from 99 to 166 questions based on responses provided. Referrals were automatically generated. This format was highly acceptable to most families. 0-6 16  
Housing instability 
Parental employment 
Household income 
Insurance status 
Problems receiving health care 
Intimate partner violence 
Housing conditions 
iScreen Computer, face-to-face, or phone interviews, 10 min, 23 items assessed via Likert scale Economic instability Informants using the computer-based platform disclosed more needs, both in quantity and as a higher degree of endorsement than those performed face-to-face. 0-18 17,18  
Concerns about lack of child care or services 
Physical or mental health coverage 
Concerns about tobacco smoke or physical activity 
Concerns about housing or transportation 
Immigration status 
Drug or alcohol problems in the home 
Family member incarceration 
Violence toward the child in the home 
Addressing Social Key Questions for Health Tool Paper and pencil, 4-5 min, 13-question screen Food insufficiency Addresses ACEs and unmet social needs but low capture rate of ACEs. 0-18 19  
Housing insecurity 
Need for legal aid 
Parental education and employment 
Child witnessed violence and bullying 
Child physical and sexual abuse 
Child separation from caregiver 
Parental mental illness or substance abuse 
WE CARE Survey Instrument Paper and pencil, 4-5 min, 6 questions Food insufficiency  0-10 20,21  
Housing instability 
Parental education and employment 
Difficulty paying bills 
Lack of child care 
Family Needs Screening Program Paper and pencil, 1-page questionnaire with mostly yes/no options Housing instability Available in multiple languages targeting recent immigrants. Resource navigators supported families following a positive screen. Electronic referrals to community-based services also provided. 0-18 22  
Difficulty with paying bills, meeting basic needs, or getting public benefits 
Need for legal aid 
Parental education 
Help with child or elder care 
Health literacy 
Experiences of discrimination 
Available social supports 
Parental alcohol, drug, and tobacco use 
Housing conditions 

Adapted from Sokol et al.23  and Moen et al.24 

NR, not reported.

If patients or families declare a need, programs should be prepared to connect families with resources, because it is unethical to screen for SDHs without providing support25 ; however, it is unclear which interventions provide the greatest benefit. For concrete needs, such as food insecurity, referring a family to a food bank may provide immediate assistance. Some needs, such as a desire for more education, may require a broader network of support to be achievable. Additionally, resources vary significantly between locations, so any program attempting to implement a screening tool for SDHs should engage members of community support organizations to review the resources available in their area. In the United States, programs that identify local resources include the United Way (www.211.org) and the EveryONE Project’s Neighborhood Navigator (www.familydoctor.org/NeighborhoodNavigator), sponsored by the American Academy of Family Physicians.

The workflow to implement SDH screening and referral also varies by clinical practice. Some clinics may have an embedded social worker, mental health counselor, or legal representative to provide direct support to patients and families who disclose need. Others clinics will need to develop partnerships with community organizations, maximizing the creation of a “health neighborhood.”25  Some interventions require additional personnel, such as a community navigator, to provide support for families18 ; however, other successful strategies have used minimal contact interventions, such as faxed referrals from clinics directly to community partners.26  How to screen patients and families is also unclear. Some studies show improved reporting with anonymous computer or web-based surveys,17  whereas others have integrated SDH screening directly into the electronic health record.27  If screening tools are to be implemented in a clinical setting, they should be implemented universally to avoid stigma and perceived bias. It is also essential to build on a family’s and community’s strengths and elicit the input of the family as to which resources may provide the most critical benefit.

Many studies on SDH screening tools have focused on implementation outcomes such as how the questions were perceived by patients or families, time spent on the screening or intervention, or quantity of referrals provided. Other studies have assessed the effect of the intervention on SDHs such as employment rates at follow-up or enrollment in social services. Although data on outcomes are mixed, they have generally yielded positive effects, and screening and interventions are acceptable to patients and families. However, few studies have quantified the impact of SDH screening and interventions on health outcomes and health care utilization.28 

Case part 2

The patient’s initial transcranial Doppler was abnormal, and she was started on monthly chronic transfusions to reduce her risk of stroke. After several months, the patient is prescribed chelation therapy with written steps for connecting with the specialty pharmacy; however, at subsequent transfusion visits, the patient’s mother reports that she has not received the medication. The team provides verbal education about iron overload; however, several months pass without the child receiving chelation, and her ferritin continues to rise. At visits, the patient’s mother understands that she is supposed to start the medication, but she is unable to articulate why she needs the medication or how to set up delivery.

Health literacy

Given the difficulty obtaining the medication, the team became concerned about the family’s health literacy, which is another factor that has been postulated to be a mediator of health disparities. Health literacy is “the degree to which individuals have the capacity to obtain, process and understand basic health information and services needed to make appropriate health decisions.”29  In the United States, risk factors for low health literacy include low socioeconomic status, racial and ethnic minority status, lower educational attainment, poor English proficiency, older age, and birth outside the country.30,31  It is estimated that 28% of parents in the United States have limited health literacy.31  Among parents of children with SCD, low health literacy has been associated with lower disease-specific knowledge, although it was not associated with increased health care utilization, such as hospitalizations or ER visits.32 

The greater the complexity of the tasks and decisions being placed before families, the greater the need for thorough understanding and comprehension, so adequacy of health literacy relies not only on the patients and families but also on the health care situation at hand. For patients with SCD receiving chronic transfusions, even those with the highest personal levels of health literacy may have difficulty with comprehension and decision making given the complex nature of the illness, the need to weigh multiple potential risks to the patient, and the many tasks set before them. In a cross-sectional study of health literacy among adult caregivers of children receiving chronic transfusion therapy and adolescents with SCD personally receiving chronic transfusion therapy, 34% of caregivers and 69% of adolescents had inadequate health literacy based on standardized testing, and low health literacy was associated with lower disease-specific knowledge. Knowledge scores for caregivers were weakly correlated with community-level median income and unemployment rates but not with insurance payor status.33  The greatest gaps in disease-specific knowledge among those caring for or receiving chronic transfusion therapy were in questions related to red blood cell alloimmunization status and risks, the availability of curative treatments for SCD, and personal indications for receiving chronic transfusion therapy, indicating opportunities for improvement.33 

One web-based decision tool that has been developed for patients with SCD considering chronic transfusion therapy or other therapeutic options is available at www.sickleoptions.org. It provides information, personal stories, and additional references as a decision aid for patients, using strategies that have been shown to overcome low health literacy. It is written at a ≤5th grade reading level and maximizes the use of graphics to improve understanding. In a randomized controlled trial, it was highly acceptable to patients and easy to use. Compared with patients who did not receive the additional decision supports, those who did had greater decisional self-efficacy, less decisional conflict, and better preparation for decision making.34 

A number of validated tools are used to assess domains of health literacy, including disease-specific literacy. See Table 2 for additional information on a sample of commonly used health literacy screening and assessment tools. Additional resources are available through the Health Literacy Toolshed, available at https://healthliteracy.bu.edu/. The majority of these tools are intended for adults or adult caregivers; however, some tools have been validated for use in children and adolescents.

Table 2.

Examples and characteristics of commonly used tools to assess health literacy

Screening toolQuestionnaires available, metrics for completionSummary of literacy domains evaluatedAdditional notesReferences
Newest Vital Sign 6 items, 3 min Written communication Available in English and Spanish. 35  
Medical numeracy, quantitation Interpretation of a nutrition label. 
Tested in children as young as 10 y old. 
Parental Health Literacy Activities Test (PHLAT) 20 items, 21 min Written communication Queries 3 domains: nutrition, growth, development; injury and safety; and medical and preventive care. 36  
PHLAT-10: 10 items, 13 min Medical numeracy, quantitation 
 
Rapid Estimate of Adult Learning in Medicine 66 items, 2-3 min Oral and written communication Subjects are graded on their ability to pronounce medical terms. 37,38  
Short Form: 7 items, 1 min Vocabulary knowledge 
 
Teen Form: 66 items, 2-3 min 
 
Teen Short Form: 10 item, 20 s 
 
Single Item Literacy Screener 1 item, 1 min Written communication Single question: “How often do you need to have someone help you when you read instructions, pamphlets, or other written material from your doctor or pharmacy?” 39  
Likert scale where 1 = Never, 5 = Always. 
Score >2 considered positive. 
Test of Functional Health Literacy in Adults 67 items, 20 min Written communication Available in English and Spanish. 40  
Short Form: 40 items, 7 min Medical numeracy, quantitation Asks questions about instructions for prescriptions or medical scenarios. Includes fill-in-the-blank portion identifying the most contextually appropriate word. 
 Validated in children 13-17 y old. 
Health Literacy Skills Instrument Computer based Print literacy Publicly available and can be self-administered using a computer. 41,42  
25 items, NR Oral literacy 
Short Form: 10 items, 10 min Numeracy 
 Internet navigation 
Screening toolQuestionnaires available, metrics for completionSummary of literacy domains evaluatedAdditional notesReferences
Newest Vital Sign 6 items, 3 min Written communication Available in English and Spanish. 35  
Medical numeracy, quantitation Interpretation of a nutrition label. 
Tested in children as young as 10 y old. 
Parental Health Literacy Activities Test (PHLAT) 20 items, 21 min Written communication Queries 3 domains: nutrition, growth, development; injury and safety; and medical and preventive care. 36  
PHLAT-10: 10 items, 13 min Medical numeracy, quantitation 
 
Rapid Estimate of Adult Learning in Medicine 66 items, 2-3 min Oral and written communication Subjects are graded on their ability to pronounce medical terms. 37,38  
Short Form: 7 items, 1 min Vocabulary knowledge 
 
Teen Form: 66 items, 2-3 min 
 
Teen Short Form: 10 item, 20 s 
 
Single Item Literacy Screener 1 item, 1 min Written communication Single question: “How often do you need to have someone help you when you read instructions, pamphlets, or other written material from your doctor or pharmacy?” 39  
Likert scale where 1 = Never, 5 = Always. 
Score >2 considered positive. 
Test of Functional Health Literacy in Adults 67 items, 20 min Written communication Available in English and Spanish. 40  
Short Form: 40 items, 7 min Medical numeracy, quantitation Asks questions about instructions for prescriptions or medical scenarios. Includes fill-in-the-blank portion identifying the most contextually appropriate word. 
 Validated in children 13-17 y old. 
Health Literacy Skills Instrument Computer based Print literacy Publicly available and can be self-administered using a computer. 41,42  
25 items, NR Oral literacy 
Short Form: 10 items, 10 min Numeracy 
 Internet navigation 

Adapted from Morrison et al.43 

NR, not reported.

Simplifying the information, using plain language, and limiting the amount of information to 3 key messages at a time are evidence-based strategies that allow families to better comprehend the critical elements being conveyed to them.43  If time allows, having patients demonstrate skills or teach back to the provider the instructions for medication or care are additional methods shown to improve comprehension and health-related outcomes. Information delivered via multiple different modalities, such as text, pictures, or video, and information that can be reviewed after the office visit have also been shown to improve understanding.43 

It is incumbent on the whole health care system to improve communication in ways that overcome low health literacy and make it easier for all patients to navigate, understand, and interpret information relevant to their health and the health of their children.43  Providers caring for patients with SCD should improve communication strategies on general topics critical for decision making and ensure that they are universally available. For patients on chronic transfusion therapy, providers should capitalize on the frequent encounters to teach and engage patients directly about their personal condition and complications so that patients can be empowered decision makers in the future.

Case part 3

The team implemented several teaching strategies, including limiting each education session to 3 critical points and providing written materials, to convey the risks of iron overload. The written information on how to set up medication delivery from the specialty pharmacy was enhanced to include specific contact numbers and expected timelines for when to hear back, as well as whom to call if medications were not delivered. Additionally, a case manager was available to reinforce that education, coordinate appointment scheduling, send reminders for medication refills and appointments, and ensure that any medications requiring prior authorizations were up to date. The patient has now been receiving regular transfusions for >18 months, and her ferritin is in the goal range. Her mother reports that the patient is “doing great.” She has not had any admissions since she started on transfusion therapy. Her mother has regularly scheduled childcare for her other children during transfusion visits because those can be planned in advance.

Quality of life and well-being

For patients with chronic illnesses, improved quality of life is arguably the most important result of any therapy; however, historically, this measure has been overlooked as an outcome. As greater emphasis has been placed on patient-reported health-related quality of life (HRQOL), generic and disease-specific measures of HRQOL have been developed and validated to measure the influence of health on functional domains, such as physical quality of life (QOL), mental QOL, fatigue, pain, social engagement, relationships, and emotional distress. Unfortunately, adult and pediatric patients with SCD report lower HRQOL in nearly every domain compared with unaffected peers, especially in the areas of pain, fatigue, and physical functioning.44  Female gender, older age, pain, socioeconomic factors such as insurance type, negative perceptions of SCD, and poor adherence to hydroxyurea increase the risk of lower reported HRQOL for patients with SCD.45-50  See Table 3 for examples of HRQOL measurement tools used for pediatric patients with SCD.

Table 3.

HRQOL measures used in studies of pediatric patients with SCD

Screening toolQuestionnaires available, metrics for completionSummary of domains evaluatedAdditional notesReferences
Child Health Questionnaire Parent-Report Form: 50 questions, 10-15 min Global general health Appropriate for parental reporting for children 5-18 y old. Child self-report validated for children ≥8-10 y old. 51,52  
 Global general behavior 1 mo recall, except change in health, which asks for 1 y recall. Responses vary from 4 to 6 level options. 
Parent-Report General health Parent report forms provide 2 summary scores for physical and psychosocial health. 
Short Form (CHQ_PF28): 28 questions, 5-10 min Bodily pain  
 Self-esteem 
Child Self-Report Form (CHQ_CF87): 87 questions, 14 min Physical functioning 
 Physical health summary 
Child Self-Report Psychosocial health summary 
Short Form (CHQ_CF45): 45 questions, 11 min Role functioning: physical 
 General behavior 
Role emotional: behavior 
Family activities 
Mental health 
Family cohesion 
Parental impact: emotional 
Parental impact: time 
Pediatric Quality of Life (PedsQL) Can be facilitated or read to patient or family member Generic Core Provides summary scores for overall, physical, and psychosocial health. 49,53  
 PedsQL Generic Core Scales: 23 items, 4 min Physical functioning Parent report and child self-report forms separated into developmentally appropriate questions available for children 2-18 y old (5-18 y old if self-reporting). 
 Emotional functioning Different referent timeframes in the SCD module, 1 mo vs 7 d (acute). 
PedsQL Generic Core Social functioning 
Short Form: 15 items School functioning  
 Sickle Cell Disease Module  
PedsQL Sickle Cell Disease Module PedsQL SCD: 43 items Pain  
 Pain-related functioning  
Pain management  
Health-related worries  
Treatment  
Communication with care providers  
Patient-Reported Outcomes Measurement Information System (PROMIS) PROMIS Parent-Report Global health measure including physical and mental health. Self-report measures appropriate for children 8-17 y old. Parent reports available for children 5-17 y old. 54-56  
PROMIS Child Self-Report Includes 22-25 domains related to physical, mental, and social health including: Shown to be responsive to acute pain in SCD. 
Physical functioning Available Proxy Profiles of varying length (25, 37, or 49 questions). 
Pain 
Fatigue 
Emotional distress – anxiety, depression, anger 
Social functioning 
Family and peer relationships 
Physical activity 
Screening toolQuestionnaires available, metrics for completionSummary of domains evaluatedAdditional notesReferences
Child Health Questionnaire Parent-Report Form: 50 questions, 10-15 min Global general health Appropriate for parental reporting for children 5-18 y old. Child self-report validated for children ≥8-10 y old. 51,52  
 Global general behavior 1 mo recall, except change in health, which asks for 1 y recall. Responses vary from 4 to 6 level options. 
Parent-Report General health Parent report forms provide 2 summary scores for physical and psychosocial health. 
Short Form (CHQ_PF28): 28 questions, 5-10 min Bodily pain  
 Self-esteem 
Child Self-Report Form (CHQ_CF87): 87 questions, 14 min Physical functioning 
 Physical health summary 
Child Self-Report Psychosocial health summary 
Short Form (CHQ_CF45): 45 questions, 11 min Role functioning: physical 
 General behavior 
Role emotional: behavior 
Family activities 
Mental health 
Family cohesion 
Parental impact: emotional 
Parental impact: time 
Pediatric Quality of Life (PedsQL) Can be facilitated or read to patient or family member Generic Core Provides summary scores for overall, physical, and psychosocial health. 49,53  
 PedsQL Generic Core Scales: 23 items, 4 min Physical functioning Parent report and child self-report forms separated into developmentally appropriate questions available for children 2-18 y old (5-18 y old if self-reporting). 
 Emotional functioning Different referent timeframes in the SCD module, 1 mo vs 7 d (acute). 
PedsQL Generic Core Social functioning 
Short Form: 15 items School functioning  
 Sickle Cell Disease Module  
PedsQL Sickle Cell Disease Module PedsQL SCD: 43 items Pain  
 Pain-related functioning  
Pain management  
Health-related worries  
Treatment  
Communication with care providers  
Patient-Reported Outcomes Measurement Information System (PROMIS) PROMIS Parent-Report Global health measure including physical and mental health. Self-report measures appropriate for children 8-17 y old. Parent reports available for children 5-17 y old. 54-56  
PROMIS Child Self-Report Includes 22-25 domains related to physical, mental, and social health including: Shown to be responsive to acute pain in SCD. 
Physical functioning Available Proxy Profiles of varying length (25, 37, or 49 questions). 
Pain 
Fatigue 
Emotional distress – anxiety, depression, anger 
Social functioning 
Family and peer relationships 
Physical activity 

Qualitative surveys have identified QOL domains that specifically affect pediatric patients receiving chronic transfusion therapy. These domains include physical and psychological aspects of pain, the positive and negative influence of chronic transfusion therapy on school and academics, fear of having a stroke, and an awareness of their disease burden and how it is influenced by their transfusion therapy.57  However, as exemplified by the patient case, pediatric patients receiving chronic transfusion therapy appear to have better HRQOL than others with SCD, despite the burdens of the treatment. As part of the Silent Cerebral Infarction Transfusion trial, parents completed a QOL questionnaire, the Child Health Questionnaire Parent Form 50, at baseline before randomization, and at the child’s last visit on the trial (36 months) or at the time of acute neurological event. There were no differences in baseline QOL scores, but at completion of the study, parents of patients on chronic transfusion therapy reported improved QOL in the domains of physical functioning, decreased pain, and improved overall health. In addition, the scores of those receiving transfusion therapy improved by a greater degree over time, reflecting improved perceptions of general health, physical functioning, and health compared with the year before.58 

Using a self-reporting tool, the Pediatric Quality of Life Sickle Cell Disease module (PedsQL SCD), pediatric patients receiving chronic transfusion therapy reported less pain and fewer effects from pain on functioning when compared with peers. Overall scores of HRQOL were significantly higher among patients on chronic transfusion therapy than among those with SCD classified as having severe disease, and patients on chronic transfusions reported significantly less pain than peers classified as having both severe or mild disease. Importantly, a higher proportion of patients receiving chronic transfusion therapy scored at a level consistent with “high functioning” in the domains of pain and pain-related functioning, reflecting the improved clinical effect of chronic transfusions.59  Despite the barriers and challenges to chronic transfusion therapy, HRQOL appears to be improved among pediatric patients who receive this life-saving treatment.

Optimizing care

Because the majority of patients with SCD in the United States are of African descent, cultural and racial barriers to care exist. SDHs and health literacy are mediators of health disparities that unjustly burden patients with SCD. Providers should be aware of the stigma that patients with SCD face and work to mitigate systemic disadvantages through resources offered to patients and through greater advocacy and policy efforts at the national level. The effects of SDH on health care utilization, disease burden, and mortality are high. For many pediatric patients with SCD, their hematologist becomes their medical home. For those on chronic transfusion therapy, the intensity of treatment may increase the burdens on an already stretched network of resources. But regular and frequent interactions with the health care system are opportunities to identify needs and refer patients and families for support. At initiation of chronic transfusion therapy, providers should screen for SDHs. More research is needed to identify ideal screening strategies, but screening tools as brief as 2 questions can predict who is at highest risk of unmet needs across multiple domains.5  Resources should be identified before implementation of screening, so that if a family screens positive, they can receive support right away. The approach to screening and referral must be tailored to the resources available, workflow in clinic, and population served, but rescreening should be planned at regular intervals because SDHs vary with time and circumstances. More research is needed to determine which interventions have the greatest impact on health outcomes; however, for the individual patient or family, asking about and providing resources for an unmet need not only builds trust in the medical team but may serve as an entry point for families to access additional support. Ideally, a multidisciplinary team that includes social work, case management, nursing, and psychology should collaborate to engage and support patients and families to achieve the best health outcomes. Clinics should adopt general strategies to overcome low health literacy in all communications with families, and implementation should be universal and culturally sensitive. Despite the many challenges posed by chronic transfusion therapy, patient QOL appears to be improved.

References

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Competing Interests

Conflict of interest disclosure: The author has no relevant conflicts of interest.

Author notes

Off-label drug use: None disclosed.

CorrespondenceJennifer Webb, Children’s National Hospital, 111 Michigan Ave NW, Washington, DC 20010; e-mail: jwebb@childrensnational.org.