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Studies at ASH Evaluate Episodic ED Utilization, Adherence, QOL in Sickle Cell Disease


Patients with sickle cell disease (SCD) typically face episodic emergency department (ED) and inpatient encounters, and innovative interventions are necessary to improve adherence to hydroxyurea treatment among youth suffering from SCD. These were the findings of 2 studies presented during an outcomes research session at the 60th American Society of Hematology Annual Meeting & Exposition.

Patients with sickle cell disease (SCD) typically face episodic emergency department (ED) and inpatient encounters, and innovative interventions are necessary to improve adherence to hydroxyurea treatment among youth suffering from SCD. These were the findings of 2 studies presented during an outcomes research session at the 60th American Society of Hematology (ASH) Annual Meeting & Exposition, being held December 1-4, in San Diego, California.

ED utilization and inpatient admission among patients with SCD is a known issue, but utilization can vary among individual patients. A 30-month study among 427 patients in 2 institutions in North Carolina, for example, found that 1 cohort of patients with SCD had more hospital admissions and ED encounters while the other cohort had more day hospital encounters.1 Pain associated with SCD is a common reason for ED visits.2,3

The first study, presented at the ASH annual meeting by Susan T Paulukonis, MA, MPH, program director, California Rare Disease Surveillance Program, evaluated both high-use and quiescent periods among patients seen in California’s non-federal hospitals over 12 years.4

“We were seeing peaks and valleys of ED utilization and we wanted to understand those patterns better,” Paulukonis said.

The data are part of the California Sickle Cell Data Collection project—a statewide effort to use a wide range of administrative, clinical, and other data sources to describe the population living with SCD, their health outcomes, and healthcare utilization patterns. Paulukonis shared analysis of data that were gathered from inpatient and ED encounters between 2005-2016 (with or without an associated inpatient stay). Patient identifiers across data set and year helped link the patient information. A true SCD case was defined as 3 or more occurrences of a SCD-specific International Classification of Disease Code (version 9 or 10) within any 5-year period between 2005-2016. Patients who met this definition and had at least a 1-year follow-up were included in the analyses.

Quiescent periods were defined as lengths of time in which a person had 0 or near 0 encounters in ED or inpatient settings. Occasional and high-use periods of ED utilization were quantitatively defined by the model.

“Among the 5090 patients that qualified for the study, the median follow-up period was 9.8 years, with a range of 1.0-11.0 years. There were over 94,000 stand-alone ED encounters, while over 59,000 ED encounters were associated with an inpatient stay,” Paulukonis said describing the cohort that was analyzed.

A 3-component model was used to combine predictive power, parsimony, and clinical relevance, including quiescent periods (mean 0.09 encounters; 88.8% of 4-week periods); occasional-use periods (mean 1.28 encounters; 10.8% of 4-week periods); and high-use periods (mean 7.48 encounters; 0.5% of 4-week periods).

All but 2 patients experienced at least 1 quiescent period during the study period, 75.9% experienced at least 1 occasional-use period, and 8.0% experienced at least 1 high-use period. According to Paulukonis, patient age did not influence the occasional- or high-use periods, which lasted a median of 8 weeks; a small fraction (3.6%) of these spells included at least some very high-use.

Younger patients (<20 years of age) had longer durations of quiescent periods (median, 24 weeks) compared with older (≥20 years) patients (median, 16 weeks).

Paulukonis concluded that most patients with sickle cell disease experience discrete periods during which ED and inpatient hospital encounters are not uncommon, separated by somewhat longer periods with few-to-no encounters. Additionally, younger patients are more likely to experience these high-frequency episodes. “We will try to tease out if, based on a patient’s status at a particular time, what will happen to the patient 2, 4, or 6 months from that time,” Paulukonis said.

Further research will include determining the likelihood of change in state for a given patient based on history and prior state. Additionally, the researchers plan to identify whether health events or patient characteristics are associated with “occasional” or “high-use” states.

Another presentation during the same session evaluated the impact of treatment adherence to hydroxyurea (HU) on the quality of life (QOL) in younger patients with SCD. Adherence issues persist among younger patients5 receiving HU for SCD, and behavioral interventions are currently being tested in the field. The study6 presented by Arlene Smaldone, PhD, CPNP-PC, CDE, Columbia University School of Nursing and College of Dental Medicine, New York, New York, and her colleagues.

The researchers examined barriers to HU adherence from the perspective of the sampled youth and their parents—poorly adherent youth between 10-18 years old, and their parents, who participated in the Hydroxyurea Adherence for Personal Best in Sickle Cell Disease (SCD), “HABIT,” trial, which had a 6-month intervention.

In addition to self-reported barriers to HU at 0, 3, and 6 months following treatment initiation, the researchers evaluated the association between adherence and generic and disease-specific health-related quality-of-life (HRQL) markers. Further, the study examined whether a community health worker could reduce adherence barriers, and if so, how.

“Adolescence is a critical time to promote self-management,” Smaldone said. “Little research has been done to identify barriers to HU use in both youth and their parents as a dyad,” she added, which they purported to evaluate with the current study.

“We used modified versions of 2 scales: the Adolescent Medication Barriers Scale (17 items; 3 subscales) and the Parent Medication Barriers Scale (16 items; 4 sub scales),” Smaldone said. Barriers reported by ≥25% of the sample were considered common, and parent proxy and youth-reported generic and disease-specific HRQL were measured at the same intervals by PedsQL and PedsQL Sickle Cell module, respectively.

The study included 28 parent-youth pairs—the median age of the youth was 14.3±2.6 years, 43% were female and 50% had Latino origins. Overall, total barriers were greater for youth (5.0±3.9) compared with parents (3.5±3.2), and a majority of parents (82.1%) parents and the youth being treated (85.7%) reported at least 1 barrier to treatment with HU.

Further, higher number of total barriers presented a significant inverse association with total generic HRQLs in both parents (r = -0.43; P = .03) and the children (r = -0.44, P <.001). A similar inverse correlation was observed for disease-specific HRQL among parents (r = -0.53; P = .005) and youth (r = -0.53; P <.001).

Smaldone highlighted some common barriers to HU that were reported by the parents who participated in the study:

  • Youth reliance on parent reminders (42.9%)
  • Adolescent frustration with living with SCD (35.7%)
  • Regimen adaptation (28.6%)
  • HU beliefs, such as parental concern with impact of HU on either fertility or the fetus (25%)

Common youth-reported barriers included:

  • Adolescent frustration (tired of living with SCD [57.1%]
  • Forgetfulness about taking HU (53.6%)
  • Tired of taking HU (39.3%)
  • Not wanting to take HU at school (28.6%)
  • Not wanting to be seen taking HU at school (25%)
  • HU ingestion issues Difficulty swallowing (25%) Taking too many pills (39.3%) Dislikes taste (35.7%)
  • Regimen adaptation (28.6%)
  • Knowledge deficits, such as not understanding how HU works (25%)

“The idea of being tired with living with the condition certainly resonated with both parents and the youth,” Smaldone said.

While it was not a statistically significant difference, parents who were a part of the group that received the health worker intervention revealed a trend in less-reported adolescent frustration. Adolescents in the intervention group had lower ingestion-related barriers over the 6-month period (-0.17 per month; P = .02). However, total barriers and other subscale scores did not significantly change over the study period, Smaldone said.

Smaldone concluded that parents and youth had varying perspectives on HU barriers, based on what was reported as a part of their study, and that improved adherence to HU treatment would require that barriers of both parents and the youth be addressed.

She noted, however, that their study was limited by a small sample size and the lack of assessment of factors such as depression/depressive systems on quality of life.

When their analysis controlled for group assignment and time, support lent by the community health workers helped youth to address HU ingestion barriers. The authors are currently conducting a multi-site trial to test the complex relationships between perceived barriers, HU adherence, and HRQL.


  1. Cline DM, Silva S, Freiermuth CE, Thornton V, Tanabe P. Emergency department (ED), ED observation, day hospital, and hospital admissions for adults with sickle cell disease. West J Emerg Med. 2018;19(2):311-318. doi: 10.5811/westjem.2017.9.35422.
  2. Glassberg JA. Improving emergency department-based care of sickle cell pain. Hematology Am Soc Hematol Educ Program. 2017;2017(1):412-417. doi: 10.1182/asheducation-2017.1.412.
  3. Cronin RM, Dorner TL, Utrankar A, et al. Increased patient activation is associated with fewer emergency room visits and hospitalizations for pain in adults with sickle cell disease [published online October 16, 2018]. Pain Med. doi: 10.1093/pm/pny194.
  4. Paulukonis ST, Roberts E, Brathwaite R, Wun T, Hulihan MM. Episodes of high emergency department utilization among a cohort of persons living with sickle cell disease. In: Proceedings from the American Society of Hematology; December 1-3, 2018; San Diego, CA. Abstract 159.
  5. Smaldone A, Findley S, Manwani D, Jia H, Green NS. HABIT, a randomized feasibility trial to increase hydroxyurea adherence, suggests improved health-related quality of life in youths with sickle cell disease. J Pediatr. 2018;197:177-185.e2. doi: 10.1016/j.jpeds.2018.01.054.
  6. Smaldone A, Manwani D, Green NS. Greater barriers to hydroxyurea (HU) associated with poorer health related quality of life (HRQL) in youth with sickle cell disease. In: Proceedings from the American Society of Hematology; December 1-3, 2018; San Diego, CA. Abstract 160.
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