Therapist-oriented home rehabilitation for adults with sickle cell anemia: effects on muscle strength, functional capacity, and quality of life.

OBJECTIVES: Considering the advances in functional rehabilitation in recent decades, therapist-oriented home rehabilitation (TOHR) has been increasingly used in the field of physical therapy because it increases patient compliance and reduces health system costs. The objective of this study was to investigate the effects of TOHR on functional capacity, muscle strength, and quality of life (QoL) in adults with sickle cell anemia (SCA). METHODS: Forty adults with SCA underwent manually guided TOHR for 12 weeks. Before and at the end of training, the following variables were assessed in the participants: distance covered in the 6-min walk test (6MWD); maximal inspiratory pressure (MIP); maximal expiratory pressure (MEP); handgrip strength (HGS); quadriceps strength (QS); and QoL using the Short Form-36 physical component summary (SF-36PCS) and the Short Form-36 mental component summary (SF-36MCS). RESULTS: After TOHR, significant increases were observed in the mean values for the 6MWD, MIP, MEP, HGS, QS, and SF-36PCS and SF-36MCS scores. The relative delta between the pre- and post-TOHR 6MWDs correlated significantly with the relative deltas of MIP (rs = 0.640, p < 0.0001), MEP (rs = 0.587, p < 0.0001), HGS (rs = 0.360, p = 0.022), and QS (rs = 0.351, p = 0.026). When the participants were separated according to their use of hydroxyurea, significant increases were observed in the relative deltas of the 6MWD, MIP and MEP values. CONCLUSIONS: This study shows that TOHR can potentially increase functional capacity, muscle strength, and QoL in adults with SCA. Furthermore, there appears to be a relationship between 6MWD gains and muscle strength gains with TOHR.Trial registration: ClinicalTrials.gov identifier: NCT04705792.

iCanCope with Sickle Cell Pain: Design of a randomized controlled trial of a smartphone and web-based pain self-management program for youth with sickle cell disease.

Many adolescents with sickle cell disease (SCD) experience recurrent and chronic pain, which has a negative impact on their health-related quality of life (HRQL). Cognitive-behavioral therapy (CBT) interventions can lead to improvement in pain and HRQL, yet due to barriers to care, most youth with SCD will not receive these interventions. To address this need for innovative programs targeting youth with SCD pain, we developed iCanCope, a tailored smartphone and web-based program that delivers a pain self-management intervention to youth with SCD. We describe the rationale, design, and implementation of a three-site parallel group randomized controlled trial with a sample of 160 adolescents with SCD and their parent caregivers. The iCanCope program includes pain self-management skills training (personalized CBT-based coping skills such as deep breathing, relaxation, and cognitive skills), goal setting, and social support. The attention control group is provided with access to a self-guided website with education about SCD. Assessments will occur at baseline (T1), immediately after completion of the intervention (12 weeks; T2) and at 6 months post-intervention (T3). Primary outcomes include coping strategies and pain intensity; secondary outcomes include physical, social, and emotional functioning, treatment satisfaction, health service use and caregiver response to youth pain behavior. Potential mediators (goal setting, self-management, and perceptions of social support) and moderators (e.g., demographic factors) will also be tested. The objective is to offer an effective, convenient, and low-cost psychosocial intervention to youth with SCD to enhance their self-management of pain.

Endurance training reduces exercise-induced acidosis and improves muscle function in a mouse model of sickle cell disease.

Sickle cell disease (SCD) mice (Townes model of SCD) presented exacerbated exercise-induced acidosis and fatigability as compared to control animals. We hypothesize that endurance training could represent a valuable approach to reverse these muscle defects. Endurance-trained HbAA (HbAA-END, n=10), HbAS (HbAS-END, n=11) and HbSS (HbSS-END, n=8) mice were compared to their sedentary counterparts (10 HbAA-SED, 10 HbAS-SED and 9 HbSS-SED mice) during two rest - exercise - recovery protocols during which muscle energetics and function were measured. In vitro analyses of some proteins involved in muscle energetics, pH regulation and oxidative stress were also performed. Exercise-induced acidosis was lower in HbSS-END mice as compared to their sedentary counterparts during both moderate (p<0.001) and intense (p<0.1) protocols. The total force production measured during both protocols was higher in trained mice compared to sedentary animals. In vitro analyses revealed that enolase/citrate synthase ratio was reduced in HbSS-END (p<0.001) and HbAS-END (p<0.01) mice compared to their sedentary counterparts. In addition, malondialdehyde concentration was reduced in trained mice (p<0.05). In conclusion, endurance training would reverse the more pronounced exercise-induced acidosis, reduce oxidative stress and ameliorate some of the muscle function parameters in SCD mice.

Evidence Supporting the Effectiveness of Transition Programs for Youth With Special Health Care Needs.

More than 90% of adolescents and young adults with chronic medical conditions will survive into adulthood. Transitioning from pediatric to adult health care services for these individuals has often times been associated with deterioration of their health and Quality of Life. Separation from their pediatric provider and lack of preparedness of the adult health care system has been identified as major barriers in preventing the successful transition of these individuals. The purpose of this review is to summarize the available data related to transitioning adolescents and young adults (AYA) with special health care needs into the adult health care system.

The Kennedy Krieger Independence Scales-Sickle Cell Disease: Executive components of transition readiness.

PURPOSE/OBJECTIVE: Youth with sickle cell disease (SCD) are at an increased risk for executive dysfunction and simultaneously have increased self-management needs compared to typical adolescents. This unique combination may contribute, in part, to difficulties during transition to young adulthood. Current measures assessing adaptive skills do not assess the executive components (e.g., initiation, prospective memory) of SCD-related self-care tasks. Modeled on the KKIS-Spina Bifida (Jacobson et al., 2013), the Kennedy Krieger Independence Scales-Sickle Cell Disease (KKIS-SCD) is a new caregiver-report measure that assesses independence with self-management of SCD-specific demands as well as routine daily activities in adolescents with SCD. Research Method/Design: Thirty-three youth with SCD and their caregivers participated in this preliminary validation study examining the construct validity of the KKIS-SCD total and composite scores (Initiation of Routines, Prospective Memory) and exploring relationships of this measure with intellectual functioning, demographic factors, illness severity, and age. RESULTS: The KKIS-SCD exhibited generally good internal consistency (Cronbach's alpha = .733 to .803), and demonstrated evidence for construct and discriminant validity when compared to an existing measure of adaptive function. The KKIS-SCD was significantly associated with caregiver-report of executive behaviors but not with intellectual functioning, demographic factors, illness severity, or age. CONCLUSIONS/IMPLICATIONS: Results provide preliminary support for the KKIS-SCD as a reliable and valid tool for the assessment of executive components of self-care management skills for youth with SCD. Identifying specific weaknesses in executive function related to self-care management skills might assist in guiding intervention and individualizing transition planning in these at-risk youth. (PsycINFO Database Record

Breathing Exercises for Inpatients with Sickle Cell Disease.

Sickle cell disease (SCD) is a painful condition wherein breathing often is compromised. This pilot study supports the premise that individuals with SCD are willing to learn breathing exercises. Medical-surgical nurses should encourage breathing exercises for managing pain and preventing complications.

Fisioterapia respiratória em crianças com doença falciforme e síndrome torácica aguda / Respiratory therapy in children with sickle cell disease and acute chest syndrome

OBJETIVO:Realizar uma revisão sistemática da literatura médica para identificar as técnicas de fisioterapia respiratória aplicadas em crianças com doença falciforme e síndrome torácica aguda, bem como descrever seu nível de evidência e recomendação. FONTES DE DADOS: Revisão bibliográfica nos bancos de dados Medline, Lilacs, SciELO e Cochrane no período de 1995 e 2009, com os descritores: "doença falciforme", "síndrome torácica aguda", "fisioterapia", "criança", "inspirometria de incentivo", em português e inglês, excluindo-se os estudos de revisão. Os artigos foram classificados por nível de evidência. SÍNTESE DOS DADOS: Foram encontrados cinco artigos; destes, três utilizaram a inspirometria de incentivo e observaram que ela evita as complicações pulmonares associadas à síndrome torácica aguda (nível de evidência II, II e IV), um deles (evidência II) comparou a inspirometria de incentivo com o dispositivo de pressão expiratória, sem diferenças entre ambos. Um artigo utilizou uma rotina de cuidados, incluindo a inspirometria de incentivo (evidência V), e observou redução do tempo de internação hospitalar e do uso de medicação oral para dor. Outro estudo com a ventilação não invasiva para crianças com desconforto respiratório e com incapacidade de realizar a inspirometria de incentivo relatou melhora da oxigenação e do desconforto respiratório (nível de evidência V). CONCLUSÕES: As técnicas de fisioterapia respiratória com dispositivos de inspirometria de incentivo, de pressão expiratória e a ventilação não invasiva podem ser aplicadas em crianças com doença falciforme e síndrome torácica aguda; o nível de recomendação é C.

OBJECTIVE:To systematically review the medical literature to identify chest physiotherapy techniques applied to children with sickle cell disease and acute chest syndrome, and to report their level of evidence and recommendation. DATA SOURCE: A bibliographic search of published articles found in Medline, Lilacs, SciELO and Cochrane databases, between 1995 and 2009, was carried out using the following keywords: "sickle cell disease", "acute chest syndrome", "physical therapy", "child", "incentive spirometry", in English and Portuguese; all review studies were excluded. The recovered studies were then classified according to their level of evidence and recommendation. DATA SYNTHESIS: Five papers were retrieved. Among them, three used incentive spirometry that played an important role in the prevention of pulmonary complications associated with acute chest syndrome (evidence levels II, III and IV); one of these studies (evidence II) compared incentive spirometry versus positive expiratory pressure and did not find differences between them. One paper reported a clinical bundle to improve the quality of care, including incentive spirometry (evidence level V). Incentive spirometry was associated with shorter length of stay and less requirement of oral pain medications. Another study evaluated the effect of non-invasive ventilation on respiratory distress in children that could not perform incentive spirometry and reported improvement in the oxygenation and in the respiratory distress (evidence level V). CONCLUSIONS: Physiotherapy techniques with incentive spirometry device, positive expiratory pressure and non-invasive ventilation can be performed in children with sickle cell disease and acute chest syndrome, with a C recommendation level.

[Problems of rehabilitation in patients with sickle cell disease in Cotonou, Benin]. / Problèmes de rééducation posés par la drépanocytose à Cotonou.

BACKGROUND: Manifestations of sickle cell disease (SCD) are polymorphous from childhood to adulthood. OBJECTIVE: The purpose of this study was to assess outcomes of rehabilitation in the patients with SCD. METHOD: This retrospective analytical descriptive study included 160 SCD patients rehabilitated from 1998 to 2006 at the National University Hospital in Cotonou, Benin. RESULTS: Mean patient was 31 years (range, 3 to 73 years). There was a female predominance (62.5%) with a sex ratio of 0.6. The type of SCD was HbAS in 53.1% of patients, HbSS in 21.3%, HbSC in 16.9%, and HbAC in 8.1%. The main clinical manifestations were classified as ischemic/infectious in 40% of patients, rheumatologic in 40%, and neurologic in 10%. Ischemic manifestations were observed in all patients with HbSS, HbSC, and HbAS while manifestations were preferentially neurological in patients with HbSS (p = 4,43.10(-3)) and rheumatologic in patients with HbAS (p<10(-3)). At the end of rehabilitation, persistent deficiencies, disabilities and limitations involved pain in 43.8% of patients, articular stiffness in 43.8%, muscular weakness in 46.9%, gait anomalies in 33.1%, amyotrophy in 21.2%, shortening of lower extremity in 16.9%, and tilting of the pelvis in 6.3%. CONCLUSION: This study shows that SCD is a highly debilitating disease. Although rheumatologic manifestations are not specific to SCD, the other complications described including femoral head necrosis, osteomyelitis, and stroke have been extensively documented in the literature. If primary prevention is unfeasible, early multidisciplinary management appears to be the most effective approach to reducing SCD-related disability.

Benefits of kinesiotherapy and aquatic rehabilitation on sickle cell anemia. A case report.

The process of hemoglobin polymerization and the consequent sickling of red blood cells that occurs in patients with sickle cell disease shortens the half-life of red blood cells. It causes vaso-occlusive complications, as well as pain and pulmonary and cardiovascular dysfunction. We evaluated an aquatic rehabilitation program used for patients with sickle cell anemia and examined the possible benefits that exercise in warm water has for the circulatory system, for relieving pain, and for increasing lung capacity. The patient was a 32-year-old female. The parameters that we used in this study include respiratory muscle strength (which was calculated by measuring maximum inspiratory pressures and maximum expiratory pressures), the McGill and Wisconsin pain questionnaires (in order to evaluate the patients' characterizations and descriptions of their pain), and the SF-36 Health Survey. The treatment included warm water exercises, stretching, aerobic exercise, and relaxation, during two sessions of 45 min per week for 5 weeks. The patient experienced a significant decrease in pain, a significant increase in the strength of respiratory muscles, and improved quality of life. We conclude that aquatic rehabilitation can be used to improve the clinical condition of sickle cell anemia patients, and we encourage more research on this new treatment regime, in comparison with other types of therapies.

Live birth after ovarian tissue autograft in a patient with sickle cell disease treated by allogeneic bone marrow transplantation.

OBJECTIVE: To report the first case of restoration of ovarian activity and live birth after cryopreserved ovarian tissue autograft in a patient without cancer treated by allogeneic bone marrow transplantation. DESIGN: Case report. SETTING: University hospital. PATIENT(S): One woman with homozygous sickle cell anemia. INTERVENTION(S): An orthotopic autotransplantation of ovarian cortical strips was performed after freeze-thawing. MAIN OUTCOME MEASURE(S): Cryopreservation of ovarian tissue, bone marrow transplantation, ovarian autograft, and restoration of ovarian function. RESULT(S): In autumn 2005, biopsy samples of ovarian tissue were cryopreserved before chemotherapy followed by bone marrow transplantation. In spring 2008, because the patient had been menopausal for 2.5 years as a result of the conditioning therapy, an orthotopic autotransplantation of thawed ovarian cortex was performed. The patient conceived spontaneously in a natural cycle in autumn 2008, and delivered a healthy female child in June 2009. CONCLUSION(S): Cryopreservation of ovarian tissue with subsequent autotransplantation is an emerging procedure for preserving the fertility of young patients with a high risk of premature ovarian failure (POF) resulting from gonadotoxic treatment. This case opens up new perspectives in cases of nonmalignant diseases.

Correlates of pain-rating concordance for adolescents with sickle cell disease and their caregivers.

OBJECTIVES: As sickle cell pain in children and adolescents with sickle cell disease (SCD) is managed primarily at home, understanding convergence of caregiver and youth pain reports may contribute to improvements in home pain management. The goal of this study was to examine concordance in pain ratings for a sample of 53 adolescents with SCD and their caregivers and to assess sociodemographic and psychosocial factors associated with concordance. METHODS: Adolescents and caregivers completed a paper-and-pencil retrospective pain questionnaire and also SCD knowledge and disease management self-efficacy questionnaires. In addition, adolescents completed a SCD pain-coping questionnaire and a performance-based measure of cognitive functioning. RESULTS: Although Pearson correlations supported convergence across measures, intraclass correlation coefficients suggested only moderate consistency in pain ratings. Significant variation in interference with daily activities (caregivers higher) and pain descriptors (adolescents higher) was identified. Few correlates were supported; SCD knowledge and disease self-efficacy were identified in correlation analyses, but younger age, higher income, and increased adolescent disease self-efficacy were the strongest independent predictors of concordance in regression analyses. DISCUSSION: Efforts to improve home management of pain in SCD should incorporate programming designed to increase parent-child communication about pain and pain interference, confidence in disease management abilities, and improved knowledge of SCD and its care, particularly for families of older adolescents.

Need for cognitive rehabilitation for children with sickle cell disease and strokes.

Stroke is a significant cause of morbidity among children with sickle cell disease (SCD). Approximately 30% of children with sickle cell anemia will have either an overt stroke or silent infarct (an injury to the brain that does not have any focal neurological findings or a history of focal neurological deficits). Despite the strong association between stroke and cognitive deficits and poor educational attainment, few interventions have been developed to address this vulnerable population of children. A cognitive rehabilitation program was piloted to improve the memory and educational achievement of children with sickle cell disease and stroke. While the intervention was feasible, additional research is needed to establish efficacy. Ongoing studies are designed to improve the cognitive impairment for children with SCD and stroke. The health and educational systems will need to work as partners to improve the cognitive and educational outcomes of these children after they suffer from stroke.

Neurocognitive sequelae of pediatric sickle cell disease: a review of the literature.

This literature review summarizes all studies relating neuropsychological performance to neuroimaging findings in pediatric sickle cell disease (N=28; published 1991-2005). Although inconsistencies exist within and across domains, deficits in intelligence (IQ), attention and executive functions, memory, language, visuomotor abilities, and academic achievement have been identified. Overall neurocognitive compromise was revealed to be related to the level of neurological injury and the location of silent infarct. Attentional and executive dysfunction is prevalent and related to frontal lobe abnormalities.

CBT in paediatric and adolescent health settings: a review of practice-based evidence.

Cognitive Behavioural therapy (CBT) has strong theoretical underpinnings that facilitate the systematic evaluation of outcomes and process of change adults. CBT has been extensively adapted for use with children and young people with session content and method of delivery modified to acknowledge developmental stage and ability. Current approaches emphasise the psychological management of the impact of symptoms of particular types of physical health difficulties and prevention of the development of psychological difficulties, as well as in the alleviation of procedurally related stress. The need for collaboration with families and other parts of a child's network is particularly relevant in the paediatric setting. This review describes what we have found helpful in our work and provides a road map of where to go to find out more about how to do more. General CBT approaches are described as well as examples of how CBT has been used specifically for procedural distress, diabetes, sickle cell disease, chronic pain and chronic fatigue.

Sickle cell disease -- when opioids and physicians fail.

An interview with a 32-year-old male with sickle cell anemia and multiple sequential admissions for vaso-occlusive crises, receiving high dose narcotic analgesics, is presented. The subsequent clinical discussion outlines psychiatric, psychosocial and treatment issues. Management of acute vaso-occlusive crisis is summarized along with a discussion of the value of comprehensive care for sickle cell disease patients. This article will be useful to physicians and consultation-liaison psychiatrists treating patients with sickle cell disease as well as policy makers developing service delivery models for this population.

Attention deficits in children with sickle cell disease.

Previous research has suggested that children with sickle cell disease may exhibit cognitive deficits even in the absence of direct cerebrovascular involvement (stroke). This study was designed to assess specific attentional deficits in children with sickle cell disease. 12 children with sickle cell disease (Hb SS) with a prior history of stroke, 14 children with sickle cell disease (Hb SS) without evidence of stroke, and 13 similar aged siblings (Hb AA or Hb AS) were compared on measures of attention, intellectual functioning, achievement, and adaptive functioning. Significant differences were found between children with sickle cell disease (with or without stroke) and healthy controls on a timed test of visual scanning, the Coding subtest of the Wechsler Intelligence Scale for Children-Revised, and subtests of Reading, Arithmetic, and Spelling from the Wide Range Achievement Test-Revised. The differences between children with sickle cell disease and their healthy siblings appear to be the result of strokes rather than sickle cell disease itself as children with sickle cell disease without strokes did not significantly differ from controls. Implications for the effects of sickle cell disease and stroke on academic performance are discussed.

Stereotypic movement disorder after acquired brain injury.

Stereotypic movement disorder (SMD) consists of repetitive, non-functional motor behaviour that interferes with daily living or causes injury to the person. It is most often described in patients with mental retardation. However, recent evidence indicates that this condition is common among otherwise normal individuals. This case study describes a patient with new-onset SMD occurring after subdural haematoma and brain injury. SMD has rarely been reported after acquired brain injury, and none have documented successful treatment. The current psychiatric literature regarding neurochemistry, neuroanatomy, and treatment of SMD are reviewed with particular application to one patient. Treatment options include serotonin re-uptake inhibitors, opioid antagonists and dopamine antagonists. SMD has been under-appreciated in intellectually normal individuals, and may also be unrecognized after brain injury. Further investigation is needed in this area, which may benefit other individuals with SMD as well.

Use of focus groups for pain and quality of life assessment in adults with sickle cell disease.

This study assessed the effectiveness of using focus groups to obtain information about the characteristics of pain and quality of life in adults with sickle cell disease and their families. Five focus group sessions were held. Four groups consisted of adults diagnosed with sickle cell disease and one was composed of family members. Although focus groups were useful for addressing the purposes of the study, several volunteers in the patient group were unable to attend due to the occurrence of pain episodes. Adults with sickle cell disease identified recurring disabling pain and its consequences as interfering with their physical, emotional, and social quality of life. Several reported a pain aura that signaled the initiation of a painful episode. Useful self-care techniques and coping strategies were also identified. Common emotional responses included anger, hostility, depression, disenfranchisement, death anxiety and fatalism. The belief by those with sickle cell disease that health professionals viewed them as drug dependent often fueled angry and hostile responses. Religion was a major source for coping. Family members' quality of life was affected and they felt the negative impact of sickle cell disease on family relationships. Family members also reported feelings of helplessness, guilt, and parental self-blame. Health care providers could use information gained through this study to positively influence the care of adults with sickle cell disease.

An intervention to increase coping and reduce health care utilization for school-age children and adolescents with sickle cell disease.

The purpose of this study was to examine whether coping with pain changed immediately and one year after a self-care intervention for school-age children and adolescents with sickle cell disease (SCD). Sixty-five children and 32 adolescents attended an educational program for living with SCD. They were then randomly assigned to relaxation, art therapy or attention-control groups. Coping was measured before, after the intervention, and 12 months later. Thirty-three children and 14 adolescents completed the one year follow-up. Although there was no significant increase in the overall number of coping strategies school-age children and adolescents used from baseline to 12 months, there was a significant increase in those strategies specifically targeted by the intervention. For adolescents, there was a significant increase in the total number of coping scores used before the intervention and one year later. When compared to well African-American adolescents, overall coping scores in this sample were significantly lower. Health care utilization related to clinic visits, emergency department visits and hospitalizations decreased significantly for all participants in the study.

Effect of transfusion in acute chest syndrome of sickle cell disease.

OBJECTIVE: To study the effects of transfusion on the clinical course and oxygenation indexes of children with sickle cell disease and acute chest syndrome. METHODS: During a 2-year period, 36 children with sickle cell disease admitted with a total of 40 episodes of acute chest syndrome were examined. Patients were given a clinical severity score indicative of the degree of respiratory distress. Arterial blood gas values were determined 4 to 24 hours before and 12 to 24 hours after transfusion, and indexes of oxygenation were calculated; six patients who were not given transfusions also had blood gases measured on admission and approximately 24 hours later for comparison. RESULTS: Blood transfusion was administered during 27 episodes (67.5%); 20 children received a simple packed cell transfusion, four had a partial packed cell exchange transfusion, and three had a simple transfusion followed by whole blood exchange transfusion because of worsening clinical symptoms. Although there was no significant change in oxygenation indexes for the six patients not treated with transfusion, there was significant improvement in all indexes after transfusion. The transfused group had more severe disease on admission, but there was no significant difference in duration of fever, tachypnea, retractions, or hospital stay between the transfusion and the nontransfusion groups. CONCLUSION: Blood transfusion, even simple transfusion of packed erythrocytes, significantly improves oxygenation in children with acute chest syndrome and is a valuable adjunct to therapy.