Coping with paediatric illness: Child’s play? Exploring the effectiveness of a play- and sports-based cognitive behavioural programme for children with chronic health conditions

Little is known about how play affects the development of children with a chronic condition. Studying play poses major methodological challenges in measuring differences in play behaviour, which results in a relative scarcity of research on this subject. This pilot study seeks to provide novel directions for research in this area. The effectiveness of a play- and sports-based cognitive behavioural programme for children (8–12 years) with a chronic condition was studied. The children and parents completed a battery of measurement tools before and after the programme. Moreover, the application of automated computer analyses of behaviour was piloted. Behaviour (Child Behavior Checklist) seemed to be positively affected by the programme. An increase in psychological well-being was observed (KIDSCREEN). Perceived competence (Self-Perception Profile for Children) and actual motor competence (Canadian Agility and Movement Skill Assessment) did not show any positive trends. These results of 13 participants suggest that children might learn to better cope with their illness by stimulating play behaviour. For the analysis of the effectiveness of programmes like this, we therefore propose to focus on measuring behaviour and quality of life. In addition, pilot measurements showed that automated analysis of play can provide important insights into the participation of children

The RESISTANT study (Respiratory Muscle Training in Patients with Spinal Muscular Atrophy): study protocol for a randomized controlled trial

Background: Spinal Muscular Atrophy (SMA) is characterized by progressive and predominantly proximal and axial muscle atrophy and weakness. Respiratory muscle weakness results in impaired cough with recurrent respiratory tract infections, nocturnal hypoventilation, and may ultimately lead to fatal respiratory failure in the most severely affected patients. Treatment strategies to either slow down the decline or improve respiratory muscle function are wanting. Objective: The aim of this study is to assess the feasibility and efficacy of respiratory muscle training (RMT) in patients with SMA and respiratory muscle weakness. Methods: The effect of RMT in patients with SMA, aged ≥ 8 years with respiratory muscle weakness (maximum inspiratory mouth pressure [PImax] ≤ 80 Centimeters of Water Column [cmH2O]), will be investigated with a single blinded randomized sham-controlled trial consisting of a 4-month training period followed by an 8-month open label extension phase. Intervention: The RMT program will consist of a home-based, individualized training program involving 30-breathing cycles through an inspiratory and expiratory muscle training device. Patients will be instructed to perform 10 training sessions over 5–7 days per week. In the active training group, the inspiratory and expiratory threshold will be adjusted to perceived exertion (measured on a Borg scale). The sham-control group will initially receive RMT at the same frequency but against a constant, non-therapeutic resistance. After four months the sham-control group will undergo the same intervention as the active training group (i.e., delayed intervention). Individual adherence to the RMT protocol will be reviewed every two weeks by telephone/video call with a physiotherapist. Main study parameters/endpoints: We hypothesize that the RMT program will be feasible (good adherence and good acceptability) and improve inspiratory muscle strength (primary outcome measure) and expiratory muscle strength (key secondary outcome measure) as well as lung function, patient reported breathing difficulties, respiratory infections, and health related quality of life (additional secondary outcome measures, respectively) in patients with SMA. Discussion: RMT is expected to have positive effects on respiratory muscle strength in patients with SMA. Integrating RMT with recently introduced genetic therapies for SMA may improve respiratory muscle strength in this patient population. Trial registration: Retrospectively registered at clinicaltrial.gov: NCT05632666

Natural history of lung function in spinal muscular atrophy

BACKGROUND: Respiratory muscle weakness is an important feature of spinal muscular atrophy (SMA). Progressive lung function decline is the most important cause of mortality and morbidity in patients. The natural history of lung function in SMA has, however, not been studied in much detail. RESULTS: We analysed 2098 measurements of lung function from 170 treatment-naïve patients with SMA types 1c-4, aged 4-74 years. All patients are participating in an ongoing population-based prevalence cohort study. We measured Forced Expiratory Volume in 1 s (FEV1), Forced Vital Capacity (FVC), and Vital Capacity (VC). Longitudinal patterns of lung function were analysed using linear mixed-effects and non-linear models. Additionally, we also assessed postural effects on results of FEV1 and FVC tests. In early-onset SMA types (1c-3a), we observed a progressive decline of lung function at younger ages with relative stabilisation during adulthood. Estimated baseline values were significantly lower in more severely affected patients: %FEV1 ranged from 42% in SMA type 1c to 100% in type 3b, %FVC 50 to 109%, and %VC 44 to 96%. Average annual decline rates also differed significantly between SMA types, ranging from - 0.1% to - 1.4% for FEV1, - 0.2% to - 1.4% for FVC, and + 0.2% to - 1.7% for VC. In contrast to SMA types 1c-3a, we found normal values for all outcomes in later-onset SMA types 3b and 4 throughout life, although with some exceptions and based on limited available data. Finally, we found no important differences in FVC or FEV1 values measured in either sitting or supine position. CONCLUSIONS: Our data illustrate the longitudinal course of lung function in patients with SMA, which is characterised by a progressive decline in childhood and stabilisation in early adulthood. The data do not support an additional benefit of measuring FEV1 or FVC in both sitting and supine position. These data may serve as a reference to assess longer-term outcomes in clinical trials

Natural history of respiratory muscle strength in spinal muscular atrophy: a prospective national cohort study

BACKGROUND: Respiratory complications are the most important cause of morbidity and mortality in spinal muscular atrophy (SMA). Respiratory muscle weakness results in impaired cough, recurrent respiratory tract infections and eventually can cause respiratory failure. We assessed longitudinal patterns of respiratory muscle strength in a national cohort of treatment-naïve children and adults with SMA, hypothesizing a continued decline throughout life. METHODS: We measured maximal expiratory and inspiratory pressure (PE max and PI max), Sniff Nasal inspiratory pressure (SNIP), peak expiratory flow (PEF), and peak cough flow (PCF) in treatment-naïve patients with SMA. We used mixed-models to analyze natural history patterns. RESULTS: We included 2172 measurements of respiratory muscle function from 80 treatment-naïve patients with SMA types 1c-3b. All outcomes were lower in the more severe phenotypes. Significant differences in PEF were present between SMA types from early ages onwards. PEF decline was linear (1-2%/year). PEF reached values below 80% during early childhood in types 1c-2, and during adolescence in type 3a. PE max and PI max were severely lowered in most patients throughout life, with PE max values abnormally low (i.e. < 80 cmH 2O) in virtually all patients. The PE max/PI max ratio was < 1 throughout life in all SMA types, indicating that expiratory muscles were most affected. All but SMA type 3b patients had a lowered PCF. Patients with types 2b and 3a had PCF levels between 160 and 270 L/min, those with type 2a around 160 L/min and patients with type 1c well below 160 L/min. Finally, SNIP was low in nearly all patients, most pronounced in more severely affected patients. CONCLUSIONS: There are clear differences in respiratory muscle strength and its progressive decline between SMA types. We observed lower outcomes in more severe SMA types. Particularly PEF may be a suitable outcome measure for the follow-up of respiratory strength in patients with SMA. PEF declines in a rather linear pattern in all SMA types, with clear differences at baseline. These natural history data may serve as a reference for longer-term treatment efficacy assessments

A systematic approach to interpreting the cardiopulmonary exercise test in pediatrics

The use of cardiopulmonary exercise testing in pediatrics provides critical insights into potential physiological causes of unexplained exercise-related complaints or symptoms, as well as specific pathophysiological patterns based on physiological responses or abnormalities. Clinical interpretation of the results of a cardiopulmonary exercise test in pediatrics requires specific knowledge with regard to pathophysiological responses and interpretative strategies that can be adapted to address concerns specific to the child’s medical condition or disability. In this review, the authors outline the 7-step interpretative approach that they apply in their outpatient clinic for diagnostic, prognostic, and evaluative purposes. This approach allows the pediatric clinician to interpret cardiopulmonary exercise testing results in a systematic order to support their physiological reasoning and clinical decision making

Organized Sports Activities Are Safe for Children With Sickle Cell Disease : A Pilot Intervention Study

Historically, children with sickle cell disease (SCD) are advised to refrain from sports activities, based on the assumption that physical exercise can trigger vaso-occlusive episodes. This pilot intervention study examined the safety (ie, no vaso-occlusive episodes) of a 10-week organized sports program for children with SCD. Eight children with SCD (5 boys/3 girls), aged 7 to 12 years old, received 10 training sessions (each 90 min) once a week. Training sessions were performed by a professional soccer club under the supervision of a medical team from the Wilhelmina Children's Hospital. During the study period, one child experienced a vaso-occlusive crisis, which could not be directly related to the organized sports program. None of the other children experienced vaso-occlusive episodes. The results of this study indicate that children with SCD can participate safely in moderate-intensity organized sports activities when personalized medical background and practical training information is shared with the trainer beforehand. All children continued their sports participation after the study period

Surgery: Moving People, Improving Outcomes

Surgery aims to improve a patient’s medical condition. However, surgery is a major life event with the risk of negative consequences, like peri- and postoperative complications, prolonged hospitalization and delayed recovery of physical functioning. One of the major common side effects, functional decline, before (in the “waiting” period), during and after hospitalization is impressive, especially in frail people. Preoperative screening aims to identify frail, highrisk patients at an early stage, and advice these high-risk patients to start supervised preoperative home-based exercise training (prehabilitation) as soon as possible. Depending on the health status of the patient and his/her outcomes during the screening and the type of surgery, prehabilitation should focus on respiratory, cardiovascular and/or musculoskeletal parameters to prepare the patient for surgery. By improving preoperative physical fitness, a patient is able to better with stand the impact of major surgery and this will lead to a both reduced risk of negative side effects and better short term outcomes as a result. Besides prehabilitation hospital culture and infrastructure should be inherently activating so that patients stay as active as can be, socially, mentally and physically. In the first part of this chapter the concept of prehabilitation and different parameters that should be trained will be described. The second part focuses on the “Better in, Better out” (BiBo™) strategy, which aims to optimize patient’s pre-, peri- and postoperative physical fitness. Prehabilitation should comprise “shared decisions” between patient and physical therapist regarding experience and evidence based best options for rehabilitation goals, needs, and potential of the individual patient and his/her (in) formal support-system. Next, a case will describe the preoperative care pathway. This chapter will close with conclusions about how moving people before and after surgery will improve their outcomes

Optimizing perioperative physical therapy care in major elective surgery to improve surgical outcome in high-risk patients: the Better in, Better out™ concept.

Frail elderly patients undergoing major surgery experience a decrease of physical capacity due to their hospital stay and surgery. This affects the risk of postoperative complications and their performance of activities, independence and participation.The Better in, Better out™ (BiBo™) strategy was developed to reduce these risks through the optimization and professionalization of perioperative hospital care in a physically activating contex

Is Measuring Physical Literacy in School-Aged Children With Cystic Fibrosis or Congenital Heart Disease Needed?

Purpose: To explore the association between cardiorespiratory fitness and other physical literacy domains in children with cystic fibrosis (CF) or congenital heart disease (CHD). Methods: In 28 children with CF (n = 10) or CHD (n = 18), aged 7 to 11 years, cardiorespiratory fitness and the following physical literacy domains were measured: (a) physical competence, (b) motivation and confidence, (c) knowledge and understanding, and (d) daily behavior (ie, self-perceived moderate-to-vigorous physical activity [MVPA]). Results: Cardiorespiratory fitness was significantly associated with motivation and confidence and self-perceived MVPA. There were no other significant associations. Conclusions: Cardiorespiratory fitness is associated with self-perceived MVPA, motivation, and confidence in children with CF or CHD

Is Measuring Physical Literacy in School-Aged Children With Cystic Fibrosis or Congenital Heart Disease Needed?

Purpose: To explore the association between cardiorespiratory fitness and other physical literacy domains in children with cystic fibrosis (CF) or congenital heart disease (CHD). Methods: In 28 children with CF (n = 10) or CHD (n = 18), aged 7 to 11 years, cardiorespiratory fitness and the following physical literacy domains were measured: (a) physical competence, (b) motivation and confidence, (c) knowledge and understanding, and (d) daily behavior (ie, self-perceived moderate-to-vigorous physical activity [MVPA]). Results: Cardiorespiratory fitness was significantly associated with motivation and confidence and self-perceived MVPA. There were no other significant associations. Conclusions: Cardiorespiratory fitness is associated with self-perceived MVPA, motivation, and confidence in children with CF or CHD