What does shared decision making look like in natural settings? A mixed methods study of patient–provider conversations

Objective: To understand the variability and nature of shared decision making (SDM) regarding a uniform type of serious medical decision, and to make normative judgments about how these conversations might be improved. Methods: This was a mixed-methods sub-analysis of the Improving Patient Outcomes with Respect and Trust (IMPORT) study. We used the Braddock framework to identify and describe seven elements of SDM in audio-recorded encounters regarding initiation of hydroxyurea, and used data from medical records and patient questionnaires to understand whether and how these tasks were achieved. Results: Physicians covered a spectrum of SDM behaviors: all dialogues contained discussion regarding the clinical issue and the pros and cons of treatment; the patient's understanding and role were not explicitly assessed or stated in any encounter. Yet no patient agreed to start hydroxyurea who did not already prefer it. There was no uniform approach to how physicians presented risk; many concerns expressed by patients in a pre-visit questionnaire were not discussed. Conclusion: In this analysis, patients seemed to understand their role in the decision-making process, suggesting that a patient's role may not always need to be explicitly stated. However, shared decision making might be improved with more routine assessment of patient understanding and concerns. Standardized decision aids might help fully inform patients of risks and benefits

A Report from the GRNDaD Multi-site Registry for Sickle Cell Disease: Iron Overload is Under-recognized and Under-managed

Introduction: GRNDaD is a prospective registry for people with SCD that opened to enrollment in 2016. Nine comprehensive SCD centers from across the United States are currently enrolling patients. The registry includes iron status and management data, important in SCD because chronic transfusion therapy is a mainstay of prophylactic management. Each unit of transfused blood introduces approximately 250 mg of iron into the blood, which can lead to systemic iron deposition, and untreated may lead to organ dysfunction or death. Methods: GRNDaD currently contains prospective baseline and annual update information on approximately 1000 people with SCD. We analyzed ferritin levels relative to genotype, age, gender, treatment type, liver iron scan results, and chelation therapy history, using chi-squared and pearson statistics for discrete and continuous data, respectively. Results: There were 783 adults in GRNDaD who had a non-crisis ferritin level from a routine follow-up visit. Nearly 1 in 3 of all participants (n=187, 31.4%) had a baseline ferritin ≥1500 mg/dL. More than a third of that group were not on chelation, and only a quarter had imaging studies to assess iron accumulation. Ferritin levels were positively associated with liver enzymes, creatinine, and homozygous SCD phenotype. Conclusion: A significant fraction of the adult SCD population in GRNDaD is living with iron overload, and management could use vast improvement. We speculate that undertreated iron overload is probably both widespread and under-recognized. We anticipate that GRNDaD may be a model for identifying and addressing deficiencies in current clinical practices for management of SCD

Knowledge of fertility and perception of fertility treatment among adults with sickle cell disease (KNOW FERTILITY)

IntroductionThis study assessed fertility knowledge in adults with sickle cell disease using the Cardiff Fertility Knowledge Scale and Fertility Treatment Perception Survey and compared knowledge scores in respondents with sickle cell disease to previously reported unaffected cohorts.MethodsThis cross-sectional study surveyed adults over age 18 with sickle cell disease at an adult sickle cell disease center using a 35-question survey addressing infertility risk factor knowledge and perceptions of fertility treatment. Analyses included summary statistics for continuous and categorical variables, univariate linear regression, and Mann-Whitney U tests for group comparisons of Fertility Knowledge Scale scores. Fertility Treatment Perception Survey scores were measured by medians of the two positive statements and four negative statements to generate separate positive and negative treatment belief scores. Statistical significance was set at p < 0.05 for all analyses.ResultsNinety-two respondents (71 female, 21 male) with median age of 32 years (IQR: 25.0, 42.5) completed the survey between October 2020-May 2021. Sixty-five percent of respondents reported taking sickle cell disease treatment and 18% reported refusing at least one sickle cell disease treatment due to fertility concerns. The mean Fertility Knowledge Score was 49% (SD: 5.2), lower than reported in an international cohort (57% vs. 49%, p = 0.001), and higher than in a cohort of reproductive-aged Black women in the USA (49% vs. 38%, p = 0.001). Less than 50% of respondents correctly identified common infertility risk factors including sexually transmitted infections, advanced age, and obesity. Mean positive fertility perception score was 3 (IQR 3, 4), and negative fertility perception score was 3.5 (IQR 3, 4). Factors associated with agreement with negative fertility perception statements included: trying to conceive, refusing sickle cell disease treatment, and undergoing fertility treatment.DiscussionOpportunities exist to improve knowledge of infertility risk factors among adults with sickle cell disease. This study raises the possibility that nearly one in five adults with sickle cell disease refuse SCD treatment or cure due to infertility concerns. Education about common infertility risks factors needs to be addressed alongside disease- and treatment- associated fertility risks

Costs and Impact of Disease in Adults With Sickle Cell Disease: A Pilot Study

We assessed the feasibility to estimate illness burden in adults with SCD, investigated factors associated with health-related quality of life (HRQoL), and estimated societal burden. We recruited 32 participants and collected data on fatigue, HRQoL, and work productivity and activity impairment via patient survey. Health care utilization was abstracted for the 12 months before enrollment using medical chart review. Mean age was 36.7 years; 84.4% of participants had hemoglobin SS or Sβthal0 disease, and 81.3% reported chronic pain (experiencing pain on ≥3 days per week in the past 6 months). Mean EQ-5D-3L visual analogue scale score was 63.4 and the index score was 0.79. The mean fatigue score was 57.9. Higher fatigue score was correlated with lower EQ-5D index score (correlation coefficient r = −0.35; P = .049) and Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me) scores, including pain (r = −0.47; P = .006), sleep (r = −0.38; P = .03), and emotion scores (r = −0.79; P \u3c .0001). The number of hospitalizations was negatively correlated with HRQoL (all P \u3c .05). Patients who reported chronic pain had significantly lower mean ASCQ-Me sleep scores (48.3 vs 57.1; P = .04) and EQ-5D index scores (0.72 vs 0.89; P = .002) than those without chronic pain. Mean estimated annual per person costs were $51 779 (median,$36 366) for total costs, $7619 ($0) for indirect costs (estimated from lost earnings of participants), and $44 160 ($31 873) for medical costs. Fatigue, SCD complications, hospitalization, and chronic pain negatively affected HRQoL. This sample experienced a high economic burden, largely from outpatient doctor visits

Risk factors for death in 632 patients with sickle cell disease in the United States and United Kingdom

Background: The role of pulmonary hypertension as a cause of mortality in sickle cell disease (SCD) is controversial. Methods and Results: We evaluated the relationship between an elevated estimated pulmonary artery systolic pressure and mortality in patients with SCD. We followed patients from the walk-PHaSST screening cohort for a median of 29 months. A tricuspid regurgitation velocity (TRV)≥3.0 m/s cuttof, which has a 67-75% positive predictive value for mean pulmonary artery pressure ≥25 mm Hg was used. Among 572 subjects, 11.2% had TRV≥3.0 m/sec. Among 582 with a measured NT-proBNP, 24.1% had values ≥160 pg/mL. Of 22 deaths during follow-up, 50% had a TRV≥3.0 m/sec. At 24 months the cumulative survival was 83% with TRV≥3.0 m/sec and 98% with TRV47 years, male gender, chronic transfusions, WHO class III-IV, increased hemolytic markers, ferritin and creatinine were also associated with increased risk of death. Conclusions: A TRV≥ 3.0 m/sec occurs in approximately 10% of individuals and has the highest risk for death of any measured variable. The study is registered in ClinicalTrials.gov with identifier: NCT00492531

Association of Sickle Cell Trait With Chronic Kidney Disease and Albuminuria in African Americans

The association between sickle cell trait (SCT) and chronic kidney disease (CKD) is uncertain