Marsha Treadwell

Quality of Life in Patients with Thalassemia Intermedia Compared to Thalassemia Major

Abstract: The impact of thalassemia major and thalassemia intermedia and their associated complications on quality of life (QOL) is largely unknown. Determining the degree of health impairment as perceived by the patient is essential information needed to recommend suitable therapy. The objective of this study was to evaluate QOL in transfusion‐independent patients with thalassemia (non‐Tx) compared with that in transfused patients (Tx) and to identify the factors that affect QOL in thalassemia. A convenient sample of 48 thalassemia patients (29 Tx and 19 non‐Tx) with mean age of 14.6 years (SD = 7.5 years) were selected during a comprehensive visit to complete a Dartmouth Primary Care Cooperative Information Chart System (COOP) questionnaire. Patients rated QOL from excellent (1) to poor (5) on five dimensions of health status. Scores of 4 or 5 represent major limitations. These results were augmented by a brief medical history and chart review. Forty‐one percent of Tx patients and 47% of non‐Tx patients reported severe impairments in 1‐6 and 1‐2 domains, respectively. The most commonly reported affected domains were feelings such as anxiety, depression, and concern of overall health status or indications of recent deterioration in health. In contrast with previous beliefs, transfusion‐independent thalassemia patients also suffer serious impairment in QOL. Presented data suggest that all patients with thalassemia undergo QOL assessment so that interventions focused on affected domains can be implemented.

Changes in intensity, location, and quality of vaso-occlusive pain in children with sickle cell disease.

&NA; A descriptive, longitudinal design was used to examine changes in current, worst, and least pain intensity during hospitalization for a vaso‐occlusive episode in children with sickle cell disease. Other dimensions of the pain experience including location and quality were also evaluated. Children reported severe pain on the day of admission with 50% of the episodes showing a current pain intensity score of >70 and a worst pain intensity score of >80. Although both pain intensity scores demonstrated statistically significant decreases by approximately 5% over the course of the hospitalization, these decreases were not clinically significant based on the recommendations made in the American Pain Societys Guideline for the management of acute and chronic pain in sickle cell disease. In contrast to the pain intensity ratings, which did not decrease in 25% of the episodes, pain location surface area decreased in 100% of the episodes. Children described the quality of vaso‐occlusive pain using all categories of word descriptors from the adolescent pediatric pain tool. These findings suggest that pain associated with a vaso‐occlusive episode is inadequately assessed and managed during hospitalization.

A Review of the Literature on the Multiple Dimensions of Chronic Pain in Adults with Sickle Cell Disease

Sickle cell disease (SCD) is a major health care and societal problem that affects millions of people worldwide. In Nigeria, 45,000 to 90,000 babies are born each year with SCD. In the United States, SCD is the most common genetic disorder, affecting more than 80,000 people, the majority of whom are African American. Sickle cell pain is the hallmark feature of SCD. Most of the research on pain from SCD has focused on children with acute pain associated with sickle cell crisis. Consequently, very little is known about the occurrence and characteristics of chronic pain, especially in adults with SCD. Individuals with SCD who experience chronic pain are often underserved, and their pain is undertreated. This undertreatment may result in millions of dollars per year spent on emergency room visits, hospitalizations, and lost work productivity. The primary purpose of this literature review was to summarize the findings from studies that evaluated the characteristics of chronic pain in adults with SCD. Each of the studies included in this review was evaluated to determine if it provided data on the following multidimensional characteristics of chronic pain: occurrence, number of pain episodes, duration, pattern, quality, location, intensity, aggravating factors, relieving factors, and impact of pain on function. A secondary purpose was to identify gaps in knowledge and directions for future research on the multiple dimensions of chronic pain in adults with SCD.

Assessment of sickle cell pain in children and young adults using the adolescent pediatric pain tool

The objectives of this study were to describe and compare the characteristics of pain experienced by children and young adults with sickle cell disease (SCD) in inpatient and outpatient settings. The Adolescent Pediatric Pain Tool (APPT), a multidimensional self-report pain assessment, was completed by African American children and young adults (mean age 15.39 +/- 4.32) with SCD during a clinic visit (n = 52), day hospital visit (n = 29), or during the first 24 hours of an inpatient stay (n = 72). Multiple linear regression revealed that pain intensity, number of body areas with pain, and the quality of pain were related to age, sex, and care setting. Pain intensity, location, and quality were of greater magnitude than previous reports of early postoperative pain in children. Examining the specific dimensions of pain intensity, location, and quality and the influencing factors of age, sex, and care setting may lead to more effective treatments for SCD pain.

Transition from pediatric to adult care in sickle cell disease: Establishing evidence-based practice and directions for research

Transition of young adults with sickle cell disease (SCD) from pediatric to adult medical care is an important priority, given medical advances that have transformed SCD into a lifelong chronic condition, rather than a disease of childhood. Successful transfer from pediatric to adult care has its foundation in collaboration among the young adult, the family, and the health care system to support building skills in positive disease management and independent living. Systemic issues in transition from pediatric to adult care for individuals with SCD include limited access to adult providers with the skills and/or interest in caring for people with SCD; poor communication and follow-up between pediatric and adult providers; and insurance coverage and reimbursement for care coordination. Family and patient issues in transition include lack of skill development for successful transition into adulthood; absence of financial independence; fear of the unknown; and increasing morbidity with age. The design and evaluation of successful transition programming in SCD requires clarity in conceptual frameworks and consistent measurement, both before and after transfer to adult care. Strategies used by three SCD transition programs and future directions for research and program development are presented.

Management of vaso-occlusive pain in children with sickle cell disease.

Purpose A descriptive, longitudinal design was used to evaluate the pain management strategies used in children with sickle cell disease who were experiencing pain during a vaso-occlusive episode. Methods A list of the medications (name, amount, mode of delivery, and frequency) prescribed and administered for pain management for each participant was recorded on the Medication Quantification Scale Worksheet, starting from day 1 of hospitalization to the day of discharge. Children were asked once each evening to provide three separate ratings of how much the pain medication helped them during the day, evening, and night using a 0-to-10 rating scale. Results Using patient-controlled analgesia (PCA), children self-administered only 35% of the analgesic medications that were prescribed and reported little pain relief. No significant relationships were found between changes in pain relief scores and the amount of analgesics administered. Conclusions Clinicians need to monitor the amount of analgesics delivered in relationship to pain relief and assist children to titrate PCA administration of analgesics to achieve optimal pain control, or to advocate for changes in the PCA regimen when children cannot assume control of pain management.

Barriers to adherence of deferoxamine usage in sickle cell disease.

We hypothesized that child cognitive disability would be a significant risk factor for non‐adherence with home deferoxamine (DFO) administration and that a factor that would contribute to improved adherence would be sharing of responsibilities for chelation between parents and patients. We explored the influences on adherence of behavioral and psychological adjustment; family stress; perceived convenience of and satisfaction with the DFO regimen; and parent and patient knowledge about DFO.

Autonomic reactivity and clinical severity in children with sickle cell disease

Individual differences in autonomic nervous system reactivity have been studied in relation to physical and mental health outcomes, but rarely among children with chronic disease. The purpose of this study was to examine the associations among autonomic reactivity, clinical severity, family stressors, and mental health symptoms in children with homozygous sickle cell disease. Nineteen children with homozygous sickle cell disease participated in a cross-sectional study involving parent-completed measures, medical record reviews and laboratory-based measures of autonomic nervous system responses to social, cognitive, physical and emotional challenges. Autonomic reactivity was significantly associated with both clinical severity and externalizing behavior symptoms. Children with greater parasympathetic withdrawal during challenges compared to rest had significantly more severe disease (r=–0.45, p<0.05); greater sympathetic activation during challenges compared to rest was associated with more externalizing behavior symptoms (r=0.44, p<0.05). Children experiencing major family stressors had internalizing behavior symptoms but no difference in autonomic reactivity or clinical severity compared to children experiencing fewer family stressors. Individual differences in autonomic reactivity may offer a new, biologically plausible account for observed variation in painful episodes, other physical complications and behavioral symptoms among children with sickle cell disease.

Improving adherence with deferoxamine regimens for patients receiving chronic transfusion therapy.

We designed a study to obtain follow-up on behavioral aspects of compliance with home deferoxamine administration, explore social factors that might influence compliance, and evaluate the effectiveness of a pilot intervention program for patients with thalassemia or sickle cell disease who were receiving chronic transfusion therapy. Thirty-one patients between the ages of 6 and 21 years and their primary caregivers were administered a 24-hour recall Interview about home care. Fifteen went on to participate in a Desferal Day Camp, which combined educational strategies with peer support. Behavioral measures of treatment adherence were similar for most patients with sickle cell disease and thalassemia. Patient compliance with days of deferoxamine administration at follow-up was associated with initial compliance, perceived support, and patient and caregiver knowledge. Increased sharing of responsibilities for home care by patients and caregivers and caregiver knowledge were associated with lower ferritin and liver iron levels. A subsample of 3 patients who were extremely noncompliant with days of deferoxamine administration was examined separately; these patients were found to be moderately compliant with the number of hours and amount of deferoxamine administered and to share fewer home care tasks with primary caregivers. Participation in Desferal Day Camp did not result in increases in knowledge or peer support, suggesting that future interventions should focus on family support and on improving self-regulatory skills. The crucial role of collaboration among patients, families, and health care providers in developing interventions to enhance adherence was emphasized.

DETECTION AND ASSESSMENT OF STROKE IN PATIENTS WITH SICKLE CELL DISEASE: Neuropsychological Functioning and Magnetic Resonance Imaging

Sickle cell disease (SCD) is associated with increased risk of stroke and cognitive impairment. This study describes a retrospective review of 65 patients who underwent routine neuropsychological testing and MRI during treatment at a comprehensive sickle cell center. It was hypothesized that (1) children with no evidence of CVA would perform lower than expected on cognitive tasks compared to population-based normative data, (2) children with strokes and children with silent infarcts would perform lower on cognitive tasks and motor skills as compared to patients with no evidence of CVA, and (3) children with evidence of silent infarcts would perform better than children with known overt strokes. This final hypothesis has not been studied previously, as children with known overt stroke and silent infarct were grouped together. Sixty-five children with SCD who were sent for routine neuropsychological testing and brain MRI were identified via retrospective chart review. Patients had been administered neuropsychological tests to assess cognitive, executive and motor function. Brain MRI was obtained from each patient and was analyzed for evidence of cerebrovascular accident (CVA). Based on MRI analysis, 27% of patients with SCD had experienced a stroke and 13% a silent infarct. The majority (59%) of patients diagnosed with stroke or infarct sustained cortical damage to the frontal lobe. Patients with SCD and no evidence of CVA functioned normally on tests of cognitive ability and achievement, but patients with CVA displayed impairments in cognitive function and comparatively lower scores on verbal and performance scales. Neuropsychological testing can identify impairments in patients with SCD with no known cerebrovascular accident. Investigations of neurocognitive functioning will help characterize patterns of deficits and can inform the ability to implement comprehensive care strategies for patients with SCD and cognitive impairment.