Rigorous and practical quality indicators in sickle cell disease care

Although sickle cell disease (SCD) was described in the medical literature over 100 years ago, many individuals affected with this condition still do not receive high-quality care.¹  In 2010, the Patient Protection and Affordable Care Act was signed into law and ushered in a period of enhanced focus on the quality and safety of care delivered to our patients.²  Over the past 7 years, there has also been a concurrent shift, with federal agencies such as the National Institutes of Health focusing on implementation science to better understand how efficacious therapies are being translated into practice. Insurers are also exploring and testing payment and delivery system reforms, such as accountable care organizations, bundle payments, and value-based payments, as potential strategies to accomplish the triple aim of lower cost, better quality, and better health.³ 

This multipronged national focus on quality and safety requires the sickle cell community to take an introspective view and ask: Are we poised to achieve the triple aim for individuals affected with SCD? Do we have quality metrics or indicators that we feel confident can appropriately assess the care we deliver? For other chronic conditions, such as asthma and diabetes, there are widely known, standard indicators to assess the quality of care delivered.^4-6  Conversely, reviews of the existing literature clearly illustrate there are several different measure sets that have been developed and used to look at different dimensions of sickle cell care delivery, care use, patient experience, and outcomes.

How do we get to a place where we have a harmonized set of measures that clinicians, administrators, researchers, and other stakeholders can use to assess the care delivered to individuals affected with SCD? The objective of this article is to outline the current status of quality indicators of SCD, identify outstanding challenges and opportunities to a harmonized, parsimonious set of metrics for quality care, and offer ideas for pathways forward.

In 2002, the Sickle Cell Disease Newborn Screening Program, administered by the Health Resources and Services Administration (HRSA), was created by Congress with a goal “to enhance the sickle cell disease newborn screening program and its locally based outreach and counseling efforts.”⁷  In 2004, the Sickle Cell Treatment Act was signed into law and authorized the creation of the Sickle Cell Disease Treatment Demonstration Program, also administered by HRSA.⁸  This program focuses improving sickle cell prevention and treatment. Over the past 15 years, grantee teams from the Sickle Cell Disease Newborn Screening Program and Sickle Cell Disease Treatment Demonstration Program have worked to improve access, coordination, and quality of care; educate providers; and engage community-based organizations in the education and care of individuals with SCD. Recent iterations of the programs have leveraged a learning collaborative framework to accelerate improvements in several dimensions related to SCD, including transitions of care, hydroxyurea use, newborn screening and follow-up, and the provision of recommended care.⁹ 

Guidelines for the care of children and adults with SCD are given in the National Heart Lung and Blood Institute’s (NHLBI) 2014 expert panel report on evidence-based management of sickle cell disease.¹⁰  Innovations in treatment of SCD, when used consistently, have led to improved quality of life, decreased disease morbidity, and enhanced life span. Inequities in public and private fiscal support for SCD, as well as gaps in the provision of high-quality care for individuals affected with this condition, have been documented in the literature.¹  Variation exists in the delivery of recommended care such as immunizations and transcranial Doppler screening, and the use of available efficacious therapies such as prophylactic penicillin and hydroxyurea.^11-13  Studies also highlight delays in timely treatment of painful episodes in acute care settings.¹⁴  As individuals with SCD continue to live longer, we need to ensure the care delivered is consistent with the 6 aims of health care (ie, safe, effective, patient centered, timely, efficient, and equitable) and will lead to favorable health outcomes.¹⁵ 

The only way to ensure SCD care aligns with the aims of health care is to measure key processes of care.¹⁵  Quality-of-care indicators are metrics used by various stakeholders to assess quality of care delivered by health care providers and health systems. These indicators are helpful to assess performance in a specific dimension of care and identify opportunities for improvement. These indicators are typically developed through a rigorous process of measure development such as the Modified Delphi approach.¹⁶  This process consists of extensive literature review, drafting candidate indicators, and convening an expert panel to rate the indicators on validity, feasibility, and, on some occasions, importance.^17,18  Indicators are refined on the basis of the review of the expert panel until a final list of candidate indicators is identified. Measurement development teams will subsequently pilot test the indicators with representative populations through chart review or review of administrative or claims data. Candidate indicators are further refined after pilot testing and subsequently finalized for use.

In 2011, Wang et al¹⁸  published the first set of 41 quality-of-care indicators related to the treatment of children with SCD in ambulatory and inpatient settings (see Table 1 for all quality indicators ). These indicators were the starting point for assessment of the quality of sickle cell care and focused on the following domains: routine health care maintenance, subacute and acute care, and chronic care. Of the 41 indicators, the expert panel members who participated in the development of measures identified 8 indicators most likely to have a large effect on improving the quality of life and health outcomes for individuals with SCD. These indicators focused on assessment and treatment of sickle cell pain and fever, comprehensive planning, use of prophylactic penicillin, transfusion, and preparation for transition to adult care.¹⁸  The majority of the indicators were based on descriptive studies and expert opinion, whereas a smaller percentage were based on data from randomized clinical trials or nonrandomized control trials (17%), and cohort or case-control studies (32%).¹⁸ 

Since the publication of these indicators, studies have used them to examine the delivery of quality care that can improve health outcomes for patients with SCD,^19,20  but the results have been mixed. One study documented continued poor adherence to recommendations regarding immunizations against pneumococcal disease (43.5% adherent to PPV23 vaccination) and influenza (21.6% adherence), and the use of penicillin prophylaxis (18.2% adherence).¹⁹  Another study examined the time to opioid administration as an indicator for patient outcomes and found that the time to opioid treatment was not associated with admission but was associated with improvements in pain scores, length of emergency department stay, and total amount of analgesia administered.²⁰  The results of these studies have identified opportunities for future quality improvement efforts.

Subsequent sickle cell quality-of-care indicators have been developed by the Quality Measurement, Evaluation, Testing, Review, and Implementation Consortium (QMETRIC) at the University of Michigan and funded by the Pediatric Quality Measures Program, authorized by the Children’s Health Insurance Program Reauthorization Act.²¹  The QMETRIC team created 18 indicators also focused on routine health care maintenance, acute care, chronic care, and satisfaction with care. The National Quality Forum (NQF) subsequently endorsed 2 of the QMETRIC team’s measures focused on transcranial Doppler screening and antibiotic prophylaxis for pneumococcal disease.²¹  Endorsement by the NQF confirms that the measure has gone through a rigorous process of development and testing, and is important and feasible to collect. These 2 indicators are the only sickle cell–specific quality indicators that have garnered NQF endorsement.

Additional sickle cell quality indicators have been developed that also used a rigorous, modified Delphi process for indicator development. These new indicators expand on domains previously identified, as developments in guidelines and evidence-based care have expanded. The HRSA-funded Sickle Cell Disease Newborn Screening and Sickle Cell Disease Treatment Demonstration programs also used a modified Delphi process to develop indicators that addressed more specific elements of the newborn screening and follow-up process.⁹  These indicators focused on newborn screening, communication and coordination of follow-up care for both SCD and sickle cell trait, genetic counseling for expectant mothers and adolescents, patient education, and patient experience.²²  Additionally, recently published indicators focused on and expanded elements of the process of transition from pediatric to adult care. These indicators extended the previously developed indicators by engaging adult providers in the assessment of the importance and feasibility of the elements of transition of care, also through a modified Delphi process.²³  Another expert panel consisting of members of the Sickle Cell Adult Provider Network identified 9 indicators as priorities for assessing the quality of the transition from pediatric to adult care. The indicators address domains for communication between pediatric and adult providers, timing of first adult visit, patient self-efficacy, quality of life, and the patient’s trust with their adult provider.²³ 

Readmission rates have also been used as an indicator to assess the quality of care delivered during hospitalization and immediately after discharge.²⁴  Published studies have identified that individuals with SCD have higher rates of readmission within 30 days of discharge relative to other chronic conditions.^25,26  There are still concerns about using these rates as a quality indicator, given readmission is the result of a complex interplay of patient, provider, and health system factors that may not necessarily be related to a failure in the delivery of inpatient care or postdischarge care.²⁷  Nonetheless, the recent focus on increased readmission rates among individuals with SCD has identified risk factors for readmission and opportunities for improvement in discharge planning and postdischarge follow-up.^28,29 

Importantly, recent work has also focused on incorporating patient and family perspectives in assessing the quality and experience of care received by patients with SCD. Additional efforts have focused on developing sickle cell–specific, patient-reported measures of experience of care, quality of life, and health outcomes, such as the Adult Sickle Cell Quality-of-Life Measurement information system, Adult Sickle Cell Quality-of-Life Measurement Quality-of-Care Survey, Patient-Reported Outcomes Measurement Information System, and Pediatric Quality-of-Life Inventory Sickle Cell Disease Module.^30-36  These measures have primarily been used as part of research studies and pilot studies that have begun to explore the use of these measures in clinical settings. Additional work is needed to identify strategies to integrate patient-reported measures of care with process-of-care indicators to obtain a comprehensive assessment of the quality of care delivered to children and adults with SCD.

As a result of the focus in the past 15 years on the development of indicators for care, efforts have also begun to standardize the use of these indicators in research studies. The goal has been to improve accessibility, enhance data interoperability, and help investigators identify opportunities for collaborative and translational research.³⁷  In 2014, the US National Heart, Lung, and Blood Institute (NHLBI) funded an administrative supplement to select high-quality standard measures related to SCD for inclusion in the PhenX (consensus measures for phenotypes and exposures) Toolkit (https://www.phenxtoolkit.org/index.php). The Sickle Cell Disease Research and Scientific Panel was created to identify and select measures that were clearly defined, well established and with demonstrated utility, broadly applicable and generally accepted, of low burden to participants and investigators, reproducible, specific, reliable, and available; and had existing standard measurement protocols. The resulting set includes indicators on frequency of pain episodes, transfusion, and stroke, and measures that capture cardiovascular, renal and pulmonary functioning, neurology, and quality of life.

Despite efforts at consensus, there is still a need to standardize measure sets and assess across research projects and National Institutes of Health-funded programs aimed at improving quality of care. There are many reasons that contribute to this lack of alignment despite progress of measurement for other chronic conditions, as mentioned. One of the major contributors is the lack of strong support for elements of quality care based on evidence in the literature. Many of the recommendations from the NHLBI expert panel’s 2014 report on the management of SCD were based on moderate or weak evidence.^10,38  Although hydroxyurea and transfusion therapy are strongly recommended for many individuals with SCD, many of the recommendations are based on less-rigorous quality of evidence, due to the paucity of clinical trials on screening, management, and monitoring for individuals with SCD. This lack of evidence-based guidelines highlights persistent gaps in sickle cell research.

As we continue to identify innovative therapies for the treatment of SCD, focus is still needed on improving the quality, and reducing the costs, of care being provided to individuals with SCD. Additionally, to better understand how the innovative interventions have begun to move the needle on existing quality-of-care measures, there must be a simultaneous focus on the contextual factors in which this work is being conducted. In recent years, the NHLBI has developed a Sickle Cell Disease Implementation Consortium funding mechanism “to support Clinical Sites to improve the health and well-being of adolescents and adults with sickle cell disease (SCD) in the US through the development of multi-modal, multi-sector interventions aimed at improving the rate at which patients with SCD receive routine primary care.”³⁹  Using the framework of implementation science will begin to bridge the gap between the innovative interventions being developed on the frontline of SCD care, assessment of quality, and the need for evidence-based elements of high-quality care.

For a long time, there were no reliable approaches to measure quality of SCD. In the past decade, numerous measures have been developed, on the basis of varying degrees of evidence, that reflect the state of evidence for guidelines. There are many directions in which to take the existing quality indicators, including expanding to different age groups, aims of care such as safety and equity, and better understanding of contextual and environmental factors using rigorous implementation-science methods. As existing quality indicators of SCD become more aligned and widely used, there are opportunities for measure development. These include expanding and adapting measures for adults with SCD with a specific focus on routine adult health care maintenance, as well as measures focused on quality of acute care management. There is also an opportunity to examine the performance of current patient-safety measures focused on health care–associated infections, medication safety, transfusion reactions, and postsurgical-associated complications within sickle cell populations.⁴⁰ 

Although SCD disproportionately affects individuals of color, there is an opportunity to potentially stratify indicators by relevant social determinants of health (eg, race or ethnicity) to assess if the indicators demonstrate differential performance among black people and white people. It is well documented within quality-improvement research that interventions can reduce racial or ethnic disparities, maintain them, or even increase the gap while still improving quality of care across a population.^41,42  There is also a unique opportunity to understand the effect of social determinants among similar racial and ethnic groups to begin to tease out the real impact of issues like income, education, housing, and other environmental factors on health maintenance, health care use, and, ultimately, health outcomes.

Finally, research to deepen our understanding of the relationship between these heath care processes and health outcomes will identify the highest-leverage intervention points to ensure that the entire sickle cell population achieves its optimal health.

S.O.O. received support from Health Resources and Services Administration (HRSA) contract HHSH250201400026C/Sickle Cell Disease Treatment Demonstration Program.

The content of this work is solely the responsibility of the authors and does not necessarily represent the official views of HRSA. HRSA did not have any role in writing of this manuscript.

Suzette O. Oyeku, Division of Academic General Pediatrics, Children’s Hospital at Montefiore, 3411 Wayne Ave, Room 861, Bronx, NY 10471; e-mail: soyeku@montefiore.org.