Impact of a paediatric-adult care transition programme on the health status of patients with sickle cell disease: study protocol for a randomised controlled trial (the DREPADO trial).

BACKGROUND: Thanks to advancements in medical care, a majority of patients with sickle cell disease (SCD) worldwide live beyond 18 years of age, and therefore, patients initially followed in paediatric departments are then transferred to adult departments. This paediatric-adult care transition is a period with an increased risk of discontinuity of care and subsequent morbidity and mortality. During this period, the patient will have to manage new interlocutors and places of care, and personal issues related to the period of adolescence. To take into consideration all these aspects, an interesting approach is to use the whole system approach to the patient, as presented in the biopsychosocial approach. The aim of this trial is to evaluate the impact of the proposed biopsychosocial paediatric-adult transition programme. METHODS: The DREPADO study is a multicentre randomised control trial comparing a control group (Arm A) to an interventional group with a paediatric-adult transition programme based on a biopsychosocial approach (Arm B). To be included, patients should have the SS, SC, or Sß form of sickle cell disease and be aged between 16 and 17 years. The randomisation in a 1:1 ratio assigns to Arm A or B. The primary outcome is the number of hospital admissions and emergencies for complications in the index hospital, in the 2 years after the first consultation in the adult department of care. Secondary outcomes consider the quality of life, but also include coping skills such as sense of self-efficacy and disease knowledge. To provide patient and parent knowledge and coping skills, the transition programme is composed of three axes: educational, psychological, and social, conducted individually and in groups. DISCUSSION: By providing self-care knowledge and coping skills related to SCD and therapeutics, helping empower patientsin relation to pain management and emotions, and facilitating the relationship to oneself, others, and care in Arm B of the DREPADO study, we believe that the morbidity and mortality of patients with SCD may be reduced after the proposed paediatric-adult transition programme. TRIAL REGISTRATION: ClinicalTrials.gov, ID: NCT03786549; registered on 17 December 2018; https://clinicaltrials.gov/.

The impact of sickle cell disease on oral health-related quality of life.

PURPOSE: The purpose of this study was to characterize the impact of sickle cell disease (SCD) on oral health and examine its impact on quality of life. METHODS: Fifty-four study subjects were recruited from the sickle cell clinic and 52 control subjects from the adolescent medicine clinic at Nationwide Children's Hospital, Columbus, Ohio. A dental exam was performed to determine each participant's caries burden. The Child Oral Health Impact Profile survey was used to assess their oral health-related quality of life (OHRQoL). RESULTS: Most subjects in both the SCD and control groups rated their overall health and oral health as "good" or "excellent." There was no statistically significant difference in OHRQoL between these groups. Additionally, no significant relationship was found between white blood cell count, medication intake, or the number of sickle cell crises as related to the caries burden. Statistically significant differences were detected in caries burden between the control group and the sickle cell hemoglobin C disease (HbSC) group (P<.02) and between the sickle cell anemia and HbSC subjects (P=.04). CONCLUSIONS: Adolescents with sickle cell hemoglobin C disease had fewer caries than peers with sickle cell anemia or controls, though the cause of this finding is not clear.

Factors that influence parents' experiences with results disclosure after newborn screening identifies genetic carrier status for cystic fibrosis or sickle cell hemoglobinopathy.

OBJECTIVE: Newborn screening (NBS) identifies genetic carriers for sickle cell hemoglobinopathy and cystic fibrosis. We aimed to identify factors during initial NBS carrier results disclosure by primary care providers (PCPs) that influenced parents' experiences and reactions. METHODS: Open-ended responses from telephone interviews with 270 parents of carriers were analyzed using mixed-methods. Conventional content analysis identified influential factors; chi-square tests analyzed relationships between factors and parent-reported reactions. RESULTS: Parents reported positive (35%) or negative (31%) reactions to results disclosure. Parents' experiences were influenced by specific factors: content messages (72%), PCP traits (47%), and aspects of the setting (30%). Including at least one of five specific content messages was associated (p<0.05) with positive parental reactions; omitting at least one of four specific content messages was associated (p<0.05) with negative parental reactions. Parents reported positive reactions when PCPs avoided jargon or were perceived as calm. Parents reported negative reactions to jargon usage and results disclosure by voicemail. CONCLUSION: Parents identified aspects of PCP communication which influenced their reactions and results disclosure experiences. PRACTICE IMPLICATIONS: Our findings suggest ways PCPs may improve communication of carrier results. PCPs should provide specific content messages and consider how their actions, characteristics, and setting can influence parental reactions.

Behavioral and emotional problems in children with sickle cell disease and healthy siblings: Multiple informants, multiple measures.

BACKGROUND: Behavioral and emotional problems in children with sickle cell disease (SCD) may be related to disease factors, or to socio-demographic factors. The aim of this study was to investigate the prevalence of behavioral and emotional problems in children with SCD living in a Western European country, compared to healthy siblings (who were comparable in age, gender, ethnicity, and socio-economic status-SES), and to a Dutch norm population. METHODS: The Child Behavior Checklist (CBCL), Teacher Report Form (TRF) and Disruptive Behavior Disorders rating scale (DBD) were distributed among caregivers and teachers of 119 children with SCD aged 6-18 years and among caregivers and teachers of 38 healthy siblings. RESULTS: Questionnaires were returned by caregivers and/or teachers of 106 children with SCD and 37 healthy siblings. According to caregivers and teachers, children with SCD had more severe internalizing problems than healthy siblings and the norm population. According to teachers, subgroups of both children with SCD and healthy siblings had more severe externalizing problems than the norm population. Children with SCD had more difficulties than healthy siblings in terms of school functioning, showed less competent social behavior and tended to have more attention deficits. CONCLUSIONS: Children with SCD are at increased risk of developing internalizing problems as a result of their disease. Subgroups of children with SCD are at increased risk of developing severe externalizing problems, which may either be related to socio-demographic factors, or to disease factors, such as neurocognitive deficits associated with cerebral infarction.

Pica for foam rubber in patients with sickle cell disease.

We report three cases of pica for foam rubber among sickle cell disease patients. All three were African-American males, and at the times of initial presentation for the pica, two of them were 11 years of age and one was 15 years of age. In all cases, the pica reportedly had been occurring for at least several years. The foam rubber was most often obtained from furniture and mattresses, as well as from ironing-board pads, stereo speakers, and padded hair rollers. Reports from other researchers also suggest that this is not an uncommon type of pica. We discuss this problem from biologic, psychologic, and social perspectives.

Neurocognitive development of young children with sickle cell disease through three years of age.

OBJECTIVE: To determine (1) the neurocognitive development of children with sickle cell disease (SCD) from 6 months through 36 months of age, (2) the independent and combined contributions of biomedical risk and parenting risk to child neurocognitive functioning, and (3) the independent and combined contributions of biomedical risk, parent cognitive processes, and family functioning to parent adjustment. METHOD: The study sample included 89 African American children and their parents served through the Duke University-University of North Carolina Comprehensive Sickle Cell Center. Measures of cognitive and psychomotor development were obtained at 6, 12, 24, and 36 months of age, and parents completed self-report measures of the cognitive processes of daily stress and attributional style, psychological adjustment, and family functioning. RESULTS: There was no significant decrease in psychomotor functioning (PDI) over time but cognitive functioning (MDI) declined, with a significant decrease occurring between the 12- and 24-month assessment points. At 24 months, poorer cognitive functioning was associated with parenting risk, in terms of a learned-helplessness attributional style, and biomedical risk, in terms of HbSS phenotype. Levels of psychological distress within the clinical range were reported by 24% of the parents, and poorer parent adjustment was associated with high levels of daily stress, less knowledge about child development, lower expectations of efficacy, and HbSC phenotype. CONCLUSIONS: The findings indicate that young children with SCD are at risk for neurocognitive impairment and provide support for the initiation of early intervention studies to promote neurocognitive development.

[Quality of life in children with sickle cell disease in Amsterdam area]. / Kwaliteit van leven bij kinderen met sikkelcelziekte in de regio Amsterdam.

OBJECTIVE: To determine the differences in quality of life between children with sickle cell disease and healthy immigrant children. DESIGN: Descriptive, comparative. METHOD: The quality of life of children with sickle cell disease between 5 and 15 years old being treated in the Emma Children's Hospital AMC in Amsterdam, the Netherlands, was assessed by using a questionnaire for parents (TNO-AZL Children's Quality of Life Questionnaire (TACQOL) parent form) if the child was between 5 and 11 years old and a questionnaire for children (TACQOL child form) if the child was between 8 and 15 years old. The study period was April-October 1998. The questionnaires were completed by 45 (parents of) patients. The results were compared with a healthy reference group of immigrant children. Statistical analysis was performed using the Student t-Test. RESULTS: Children with sickle cell disease as well as their parents scored significantly lower on the items general physical, motor and independent daily functioning and on occurrence of negative emotions. No significance was observed for the items cognitive functioning and school performance nor for social functioning or occurrence of positive emotions. CONCLUSION: In children, sickle cell disease leads to compromised physical and possibly also psychological wellbeing, as well as the experience of decreased independence in daily functioning, but not to compromised cognitive or social aspects of the quality of life.

Quality of life in children with sickle cell disease in Amsterdam area

OBJECTIVE: To determine the differences in quality of life between children with sickle cell disease and healthy immigrant children. DESIGN: Descriptive, comparative. METHOD: The quality of life of children with sickle cell disease between 5 and 15 years old being treated in the Emma Children's Hospital AMC in Amsterdam, the Netherlands, was assessed by using a questionnaire for parents (TNO-AZL Children's Quality of Life Questionnaire (TACQOL) parent form) if the child was between 8 and 15 years old. The study period was April-October 1998. The questionnaires were completed by 45 (parents of) patients. The results were compared with a healthy reference group of immigrant children. Statistical analysis was performed using the Student t-Test. RESULTS: Children with sickle cell disease as well as their parents scored signifcantly lower on the items general physical, motor and independent daily functioning and on occurrence of negative emotions. No significance was observed for the items cognitive functioning and school performance nor for social functioning or occurrence of positive emotions. CONCLUSIONS: In children, sickle cell disease leads to compromised physical and possibly also psychological wellbeing, as well as the experience of decreased independence in daily functioning, but not to compromised cognitive or social aspects of the quality of life. (AU)

Comparison of attitudes about communication in children and adolescents with sickle cell disease.

Survey responses of 24 African-American children and adolescents with sickle cell disease suggest similar, generally positive communication attitudes.

Adolescents' perceptions of psychosocial factors related to sickle cell disease.

12 adolescents with sickle cell disease rated psychosocial reactions to their disease. They had positive social and family relationships but had concerns about body development, self-confidence, being a burden on their families, and death.

Illness specific patterns of psychological adjustment and cognitive adaptational processes in children with cystic fibrosis and sickle cell disease.

Illness-specific patterns of adjustment and cognitive adaptational process were identified in children (7-12 years of age) with cystic fibrosis (CF, n = 40) or sickle cell disease (SCD, n = 40). Anxiety diagnoses were most frequent for both illness subgroups but children with CF had a higher rate of oppositional disorder (27.5%) than did children with SCD (2.5%). Significant portions of the variance in adjustment were accounted for by stress appraisal (19%), expectations of efficacy (9%) and health locus of control (9%) for children with CF and by stress appraisal (21%) and self-worth (12%) for children with SCD. The interaction of general and specific illness tasks and adaptational process with developmental tasks in delineating intervention opportunities is discussed.

Children's perceptions of psychosocial factors related to sickle cell disease.

Few studies have examined psychosocial issues from the perspective of children who have sickle cell disease. In the present study psychosocial issues of self-esteem, social and family relationships, anxiety, depression, anger, denial, and knowledge were examined using a researcher-developed survey and 12 children with sickle cell disease as informants.

Sickle cell disease. Still a management challenge.

Early diagnosis of sickle cell disease enables physicians to initiate prompt treatment and provide appropriate education and counseling for patients and their families. In this article, Dr Burdick reviews the management of major complications of the disorder, discusses the psychosocial impact, and examines the possibilities for cure in the future.

Scholastic performance of children with sickle cell disease.

This article describes the scholastic performance of two groups of African American children with sickle cell disease: 17 with sickle cell anemia (SS), the most severe form, and 15 with sickle-C disease (SC), a less severe form. These two groups are compared to 34 healthy African American children with similar demographic characteristics. To determine scholastic performance, standardized reading and mathematics achievement test scores and school attendance were obtained. The conclusions drawn from these data reveal significant differences in reading, math, and school attendance of SCD children compared to healthy children. The healthy children's scores and attendance were better on both indicators. The most critical finding of the study shows that SC children, the ones with the less severe form of SCD, scored the poorest in reading and mathematics. As expected, the SS children had the most absences during a school year. The results of this descriptive study suggest that SCD children are at greater educational risk of school failure than their healthy peers.

Psychosocial aspects of sickle cell disease (SCD) in childhood and adolescence: a review.

This paper reviews the literature on the psychological and social aspects of Sickle Cell Disease (SCD) and discusses the clinical implications of its impact on children and their families. Sickle Cell Disease is a family of blood diseases including sickle cell anaemia (SS), SC disease (SC), and sickle B thalassaemia (SBThal). Research on the psychological and social aspects of SCD, particularly in the UK, has been limited and of varying methodological quality. The psychosocial adaptation of children and adolescents with SCD and their families has been associated with the personality and developmental stage of the child, family attitudes and behaviour, socioeconomic status, and social and environmental support. Concerns about the quality of interpersonal relationships within families have also led to investigations of family characteristics and social networks, and some research studies have pointed to different ways of coping associated with specific network and family structures.

Psychosocial and family functioning in children with sickle cell syndrome and their mothers.

OBJECTIVE: The purpose of this study was to compare the psychiatric functioning of 61 sickle cell youth and their families with nondiseased sibling controls. METHOD: Functioning assessed by multiple informants included indices of behavioral, cognitive, and family/interpersonal functioning, self-esteem, life events, coping strategies, temperament, adaptive behavior, and parental psychopathology. RESULTS: Key findings were that sickle cell patients evidenced more depressive symptoms and associated attributional style, and externalizing behavioral difficulties than did nondiseased siblings. With age, sickle cell youth evidence increasing adaptive behavior deficits and internalizing symptoms. Illness severity was related to symptoms of internalizing behavior and fewer daily living skills. Associations were found between maternal and child coping. CONCLUSIONS: It is recommended that psychiatric consultations routinely be conducted with these children, particularly at times of family stress and developmental transitions. Psychiatric interventions should focus on ameliorating emotional difficulties via enhancing adaptive coping strategies.

Social interactions between children with cancer or sickle cell disease and their peers: teacher ratings.

We evaluated the social reputation of children with cancer (nonprimary brain tumors) (n = 26) children with a primary malignancy involving the central nervous system (n = 15), and children with sickle cell disease (SCD) (n = 33) using a standardized measure of social reputation, the Revised Class Play (RCP). Each child's classroom teacher completed the RCP, an instrument designed to assess sociability-leadership, aggressive-disruptive, and sensitive-isolated interpersonal qualities. Matched-pairs analyses comparing the child with chronic illness to one peer in each class who was the same gender/race and the closest date of birth showed children with cancer were nominated more often for sociability-leadership roles and less often for aggressive-disruptive roles. Brain tumor survivors were nominated more often for sensitive-isolated roles. Children with SCD were not significantly different from peers. Findings suggest that the stressful life events associated with cancer or SCD did not have a significant negative impact on social adjustment for school-aged children with these diseases. The data demonstrate the critical need for appropriate controls when evaluating psychosocial morbidity and chronic illness. Better understanding of the processes of resilience for these children who are routinely exposed to heightened stress provides an opportunity to improve the competence of other high-risk youth.

Stress, coping, and psychological adjustment of adults with sickle cell disease.

A transactional model of psychological adjustment to chronic illness was examined with 109 African-American adults with sickle cell disease (SCD). Good psychological adjustment was associated with lower levels of perceived daily stress and stress regarding SCD illness tasks, higher efficacy expectations, less use of palliative coping methods, less use of negative thinking/passive adherence pain-coping strategies, and family functioning characterized by high levels of support and low levels of conflict and control. Overall the underlying stress and coping conceptual model accounted for 44-50% of the variance in psychological adjustment.

Sickle cell disease pain: 2. Predicting health care use and activity level at 9-month follow-up.

This study examined 9-month follow-up data obtained from adults with sickle cell disease (SCD) participating in a longitudinal study of pain-coping strategies. Of 99 subjects completing the baseline assessment of pain-coping strategies, 89 (90%) completed a structured pain interview assessing health care use and activity reduction during painful episodes over the follow-up period. Regression analyses controlling for demographics and disease severity revealed that baseline Negative Thinking and Passive Adherence was associated with greater activity reduction and more frequent health care contacts during the subsequent 9 months. Additional correlational analyses revealed that if levels of Negative Thinking and Passive Adherence increased from baseline to follow-up, this increase was associated with even further reductions in activity level during painful episodes. Changes in disease severity were not associated with any of the follow-up measures. Comparing pain-coping strategies assessed at baseline to pain-coping strategies measured at follow-up revealed that, without intervention, pain-coping strategies were relatively stable over time.