24 Respiratory Symptoms

Rose Sharpe, Megan Jordan, Raymond Barfield, Sarah Gall, Margarita Bidegain, Kristen Lakis, Renee Bartle, and Emily Layok

Introduction

Respiratory symptoms in children are common and are one of the most distressing symptoms to patients, parents, and caregivers. They occur in 25.6% of children with progressive genetic, metabolic, or neurological conditions.^1,2,4 Systemic problems, such as weakness of the respiratory musculature, are seen with Duchenne muscular dystrophy, spinal muscular atrophy, or cystic fibrosis (discussed in Chapter 31). Localized problems such as airway abnormalities, swallowing difficulties, aspiration of secretions, or pneumonia may cause breathing difficulties. Metastases of tumors can cause widespread disease in the entire thorax, pleural effusions, pneumothorax, or airway compression. As children near the end of life, it is common to see the development of abnormal breathing patterns such as apnea, dyspnea, or Cheyne-Stokes respirations and difficulties with cough and secretion management.⁵ Respiratory symptoms at the end of life tend to be one of the most distressing and leave a lasting impact on those who witness them.

Respiratory management in children is complex. There are many interventions both invasive and noninvasive that can be offered to provide support and comfort. Not being able to breathe normally is frightening to both the child and the caregiver. Care for the child will need to involve the coordinated efforts of the interdisciplinary team. The members may vary depending on the institution, but typically include a physician, nurse, social worker, child life specialist, chaplain, psychiatrist and/or psychologist, and respiratory therapist (see Table 24.1). The team collaborates to help support and empower the child and family through times that can be difficult and anxiety producing. It is important to consider not only the child and family’s physical needs, but also their spiritual and psychosocial needs.

This chapter examines common respiratory symptoms with emphasis on those seen in advanced illness including end of life, management of these symptoms by the interdisciplinary team, and how advance care planning can help ease the distress of making decisions “in the moment” when respirations can no longer be sustained outside of the intensive care unit (ICU). Discharges to palliative care or hospice do not typically occur from ICUs. However, this is becoming more common with medical interventions that extend life. Challenges related to respiratory support during the transition from the ICU to home will also be explored.

Clinical Vignette

Samuel was a 17-year-old patient with a rare, metastatic cancer called clear cell sarcoma. He was passionate about literature and theater. He loved to read and re-read the novel The Life of Pi⁶ because Pi was lost at sea in a lifeboat with a tiger and Pi masters and trains the tiger. Samuel knew what it was to be lost, but he was unable to master the tiger of his cancer. His parents were able to pay for every available treatment, including radical surgical resection of tumors in Europe. But the cancer progressed relentlessly. As he approached the end of his life, Samuel’s respiratory agony increased because of uncountable pulmonary metastases. He was in the hospital, but he frequently asked to be alone. His parents would sit outside his room, searching the internet for new treatments. His father asked the palliative care physician to visit with him, just in case there were options available to make him more comfortable. Samuel agreed to meet. The palliative care physician sat with him quietly for almost half an hour before saying anything. He finally asked, “Samuel, what do you need right now?” Speaking in broken sentences, a few words at a time, Samuel answered, “I would be so much better if I could just cry. But I cannot breathe in enough air to cry. All I want is to cry one time, and I will be OK.” The palliative care physician assembled a team that included Samuel’s nurse and respiratory therapist, and, with a combination of opioids, anxiolytics, oxygen, diuretics, and beta-agonists, Samuel was able to breathe in enough to cry. When he finished, his parents agreed to focus on comfort care. Samuel was given relief from his respiratory distress, and 2 days later he died peacefully, surrounded by people who loved him.

Dyspnea is “a subjective experience of breathing discomfort that consists of qualitatively distinct sensations that vary in intensity.”⁷ This experience is affected by physiological, psychological, social, and environmental factors and can be found in patients of all ages with a variety of illnesses. The prevalence of dyspnea in pediatric patients with life-threatening conditions varies from 21% to 80%.⁸ Dyspnea has been identified to cause a substantial or great deal of suffering by parental report in pediatric patients with cancer and heart disease at the end of life.^9,10 The causes of dyspnea (Table 24.2) vary widely and can occur due to the malformation or malfunction of different organ systems or the presence of psychological distress such as anxiety and depression.

The assessment of dyspnea in the pediatric population often includes the evaluation of both subjective and objective elements. Physical signs that may indicate the presence of dyspnea include increased respiratory rate, pursed lip breathing, gasping, and use of accessory muscles, recognized through obvious retractions.^11,12 The presence of labored breathing may not fully characterize the patient’s experience or may only occur during times of stress, such as feeding for neonates or exertion in older children and adolescents.^8,12 Self-report of this subjective sensation is often difficult in pediatrics given lack of communication skills in neonates, young children, and those with severe neurologic impairment.⁸ Therefore, assessment of dyspnea often requires collaboration with parents to assess triggers and response to exertion. Tools to measure dyspnea in children are limited and most are validated for patients older than 6 years who have cystic fibrosis. These include “modified Borg Scale,” “Visual Analog Scale,” and “Dalhousie Dyspnea Scale,” which can also be used in children with asthma. They have been found to be more effective when combining verbal and pictorial descriptions.^8,13 Dyspnea assessment, as with all symptoms in palliative care, requires careful history-taking and physical examination to determine the possible underlying etiologies of this symptom and the impact of dyspnea on the patient’s quality of life.

Modalities that target reversing or improving the underlying etiology for dyspnea are the first step in management of this symptom if these interventions are concordant with patient and family goals of care. Management for dyspnea is best provided in a calm environment, which will help minimize any associated anxiety. The treatment strategies for dyspnea are a combination of pharmacologic (Table 24.3), psychosocial, psychological, and mechanical interventions (Box 24.1) that should be escalated to match the intensity of the symptom.^11,12 Education should be provided to the patient and family about options for dyspnea management strategies, as well as consistent reevaluation of the patient’s dyspnea with each intervention.¹⁴

A Cochrane review of adults with dyspnea showed that interventions such as mechanical vibration of the chest wall, electrical stimulation of leg muscles, walking aids, and breathing training helped relieve shortness of breath; there have been mixed results for acupuncture and acupressure.¹⁵ There is insufficient evidence to assess relaxation, cognitive-behavioral strategies, and distraction for the management of dyspnea.^7,15,16 Many children respond well to deep breathing, bubbles, and use of a pinwheel to help control breathing when they are feeling anxious and short of breath. Use of a fan to direct air movement over the face has been observed to reduce dyspnea.^7,14 Appropriate positioning, especially for patients with neuromuscular disorders can help reduce dyspnea and orthopnea.

In patients with hypoxemia, use of oxygen is appropriate, but very few studies regarding this intervention have been done systematically in patients with life-threatening illnesses.⁸ A 2011 randomized control trial in adults evaluated oxygen versus medical air for non-hypoxemic palliative care patients and found that neither gas was superior in improving quality of life or relieving shortness of breath.¹⁴ Patients and families often find comfort in having access to oxygen, especially in the home setting, as this provides them a sense of self-control in managing shortness of breath. Some pediatric patients with neuromuscular weakness or airway anomalies may benefit from the use of airway clearance therapy to aid with secretion management, noninvasive ventilation (NIV), or even mechanical ventilation via tracheostomy. A conversation about goals of care should be held with the patient and family prior to initiation of these life-altering interventions. Time-limited trials of NIV are possible to assess for improvement in dyspnea. In the setting of reversible illness, some families may also consider intubation; however, the decision to discontinue ventilatory support may be more difficult for families.¹²

Opioids are the primary medication used to manage dyspnea in patients with advanced illness.¹⁷ Sometimes medical personnel and parents are fearful of respiratory depression and the possibility of accelerated death with opioid therapy. However, studies have shown improvement in respiratory rate and dyspnea control without iatrogenic hypoventilation or hypoxia. Education, careful observation, and appropriate dosing can alleviate this inappropriate fear of opioid use.^8,12,14 The typical starting dose of opioids for the management of dyspnea is one-quarter to one-half of the dose used for pain (Table 24.3), and this dose can be increased as needed for symptom relief. In adults there is evidence for use of low-dose sustained-released morphine for treatment of chronic refractory shortness of breath in the setting of advanced progressive disease.¹⁸ Oral opioid preparations are preferred to intravenous for management of chronic dyspnea.^8,12 Intranasal fentanyl is a safe and effective medication for management of acute respiratory distress in children with life-limiting conditions.¹⁹ There is no evidence to support the use of inhaled opioids.¹⁷

Benzodiazepines are not meant to serve as a stand-alone therapy to manage dyspnea in children.¹² A meta-analysis of studies assessing the use of benzodiazepines in adults with advanced illness showed no improvement in shortness of breath.²⁰ They can be used to treat comorbid anxiety, which may help improve dyspnea.^7,14 Benzodiazepines should be considered in refractory dyspnea that is not improving despite various treatments. Other medications that may also provide relief of associated symptoms include mucolytics, bronchodilators, diuretics, anticholinergics, and steroids. Steroids can be a double-edged sword, reducing airway inflammation but also carrying the risk of weakening respiratory muscles, and they should be used with caution.⁷

Child life specialists work closely with patients who have respiratory compromise secondary to chronic illnesses or nearing the end of life. They collaborate with other members of the psychosocial team, including psychiatry, psychology, chaplains, and social work to develop nonpharmacological coping and symptom management plans for these patients.²¹ Child life specialists begin relationships with patients through play to establish friendly rapport with the patient and to earn the patient’s trust, to allow assessment of the patient’s developmental stage and establish an appropriate teaching plan or intervention.

For patients with respiratory compromise an effective activity for improving lung capacity and to help with coping is blowing bubbles. Blowing bubbles has many benefits including simple stress relief, promoting normalization, supporting development, and, most importantly, helping one to breathe deeply and steadily. Bubbles are a familiar and fun visual to show a child how they can control their breathing. A child life specialist can then help the child to replicate this sensation when they are not blowing bubbles to help control their breathing.²²

Guided imagery can be extremely beneficial for patients with chronic respiratory symptoms. Not only does it encourage a patient to be distracted for a moment, but it helps to regulate their body and breathing, provides a sense of control over their body in an environment where they have little control, and allows them to better manage their distress.²² These exercises can be done with family or the patient’s nurse as well as through technology. There are a variety of applications for cellphones, computers, and tablets that promote and assist with guided imagery, guided visualizations for deep breathing, and meditation that can provide benefits to many patients.²³ Through an established trusting relationship, play, and developmentally appropriate education, child life specialists can help patients understand their breathing during challenging times.

Psychologists or psychiatrists in conjunction with the interdisciplinary team can assist patients who experience anxiety which can lead to respiratory difficulties by incorporating cognitive-behavioral therapy (CBT). CBT has been associated with improvements in quality of life in patients with anxiety.²⁴ Social workers can also assist with helping patients gain distress tolerance skills, develop relaxation techniques, and utilize guided imagery skills. The social worker is also attentive to the family system and can help them think through complex decision-making.

Some families find benefit from spiritual support. Chaplains can provide spiritual resources, assist with stress management, and can help with traditional rituals. If anxiety can’t be managed with these interventions, the addition of an anxiolytic may be helpful to manage anxious behavior leading to dyspnea. These medications can be prescribed by the psychiatrist or the patient’s primary provider.

Cough

Cough interferes with activities as basic as eating and sleeping and can also contribute to social isolation by discouraging affected individuals from engaging in public events. Moreover, cough leads to fatigue, abdominal or chest pain, and even vomiting and rib fractures. Persistent cough is a serious problem affecting quality of life that healthcare professionals sometimes underestimate. Our understanding of triggers such as afferent sensory nerves originating in the upper airway,²⁵ TRPV1 receptors and TRPA1 receptors,²⁶ has improved. Unfortunately, preliminary studies in adults have not shown clinical effectiveness of therapies targeted to these pathways.

Identification of underlying pathology is important in treating cough in those diagnosed with serious and life-threatening illnesses. Overt and silent aspirations are clear triggers of chronic cough and can be treated with pro-motility agents and thickened-consistency diets. Cough due to anatomic excitation of contributory reflexes and nerves, often found in malignancy, can sometimes be treated with surgery, chemotherapy, and radiotherapy, both external beam and brachytherapy. Many patients with life-threatening illness are also vulnerable to infection and fluid overload, which can be treated with antibiotics and diuretics, if so aligned with the patient’s and/or caregiver’s goals.

Cough may respond to N-methyl-d-aspartate (NMDA) receptor antagonists, such as dextromethorphan.²⁷ This is often available in low-dose formulations in over-the-counter cough preparations. Suppression of cough can also be achieved with systemic agents such as opioids. Recently, and especially in adults with lung cancer, there has been an increasingly recognized role of speech therapy, which teaches patients strategies to control cough, identify triggers of cough, and receive training in oral hygiene.²⁸ Even more recently, a randomized controlled trial in adults showed even greater efficacy of pregabalin and speech therapy together for chronic cough.²⁹ Very few studies in pediatric patients exist, but several studies have concluded that demulcents, particularly honey, can be useful in this population.³⁰

Secretions

Suctioning is the first line treatment for airway maintenance in children with excessive or difficult to manage secretions. In-home portable suction equipment is easily provided. Deep suctioning is thought to cause discomfort in patients; thus, this is generally avoided at end of life.

Excessive, thin secretions can be controlled by a variety of anticholinergic medications, although the evidence for these strategies is quite weak (Table 24.3).³¹ Within this class of medications, the one least likely to cross the blood–brain barrier is glycopyrrolate. Other agents commonly used to thicken or dry secretions include atropine ophthalmic drops administered orally, hyoscyamine, and scopolamine. These medications need to be carefully titrated to avoid causing excessive dry mouth and so that the secretions do not become too thick. If this complication arises, secretions may become much harder to move within the airways, leading to the formations of large, solid mucous plugs, which can then worsen the child’s respiratory symptoms. Additionally, families and caregivers also need to be alert to other anticholinergic symptoms, such as urinary retention, which sometimes requires indwelling urinary catheterization and may cause worsening constipation.

Children with sialorrhea, in particular those with neurologic disorders, may require more intensive therapies given the impact of this symptom on their quality of life. Therapies such as botulinum toxin A injections (BtA) and salivary gland surgery may be good options for intervention. Botulinum toxin A has many clinical applications but has gained popularity as a treatment for saliva management. Injections are typically administered with the use of local anesthesia or done under general anesthesia in conjunction with other procedures. A saliva control clinic in the United Kingdom found effective responses to BtA in 61% of their patients with a mean duration of efficacy of 4 months, with a range of 2 weeks to 18 months. Sixteen percent of children developed complications which included dysphagia (16%) and xerostomia (3%), and a further 4% developed thick, sticky secretions.³² Another smaller study showed that 80% of caregivers noted an improvement in the child’s quality of life after botulinum toxin (Botox) treatment of sialorrhea.³³ If all forms of pharmacotherapy including BtA are not effective, surgery can be considered especially if the child is having issues with aspiration. Salivary gland surgery, which comprises of either submandibular duct transfer with excision of sublingual glands for children not at risk for aspiration or bilateral submandibular excision with bilateral parotid duct ligation for children with known aspiration risk, was an effective intervention for sialorrhea in 88% of the children at a saliva control clinic.³²

In advanced illness, the focus is most frequently on drying secretions. There are times, however, when thinning secretions is most helpful to patients. This is often the case when patients have infection or tumor leading to cough and difficulty expectorating their mucus and secretions. In these situations, thinning or emulsifying secretions with mucolytics makes it easier for patients to expectorate (Table 24.3). Normal saline nebulizer treatments and guaifenesin therapies are most commonly used. A less common and costlier alternative is aerosolized acetylcysteine.

Pulmonary Hemorrhage

Pulmonary hemorrhage presents with a clinical spectrum ranging from blood-tinged sputum to sudden, life-threatening hemoptysis. In the pediatric population, significant pulmonary hemorrhage commonly arises from complications of advanced lung disease, sepsis, or end-stage liver disease. Bleeding is exacerbated by coagulopathies and thrombocytopenia. Pediatric oncology patients experience pulmonary hemorrhage as a result of both invasive disease and the treatment for disease, including radiation and chemotherapeutic agents such as cyclophosphamide, busulfan, and bleomycin.³⁴ Patients who have had hematopoietic stem cell transplantation are particularly vulnerable to both infectious and noninfectious causes of lung injury that can lead to intraparenchymal bleeding.³⁵ Diffuse alveolar hemorrhage in these patients has a particularly high mortality rate.³⁶ Beyond these specific causes, pulmonary hemorrhage can be seen in any critically ill patient who is mechanically ventilated. Therapies are generally directed at treatment of underlying infections, correction of coagulopathies and thrombocytopenia, and, in some cases, administration of corticosteroids, activated Factor VII, and aminocaproic acid to stabilize clots and judicious use of positive pressure in intubated patients.³⁷ But with overwhelming hemorrhage, such interventions are often inadequate to control the bleeding, and they may be inappropriate in the context of end-of-life care where the priority is comfort. Knowing the diseases and treatments associated with increased risk of bleeding can help a team create a plan in light of an individual patient’s trajectory and goals of care.

When a patient has a disease or undergoes a treatment known to increase the chance of pulmonary bleeding, anticipatory care can mitigate trauma and avoid unwanted or ineffective interventions.³⁸ Whether mild or severe, pulmonary hemorrhage can be frightening to patients, families, and healthcare professionals. The possibility of hemorrhage and the plan for intervening should be explained to the patient, if appropriate, based on age and maturity, and to the patient’s family and other care providers. The visual impact of bleeding can be profound. To diminish this, patients can wear darker clothing, and darker bed linens and towels can be provided. When patients are alert, pulmonary hemorrhage can cause distress. As with other causes of dyspnea, respiratory distress associated with bleeding can be eased with opioids. During an acute event, a bolus of midazolam given subcutaneously or intravenously can relieve anxiety and reduce a patient’s memory of the event.³⁹ If the bleeding persists, the patient may benefit from a continuous midazolam infusion. It will be important for patient and family to be counseled that this infusion will cause moderate sedation and relief of anxiety for the patient while the medical team continues to manage dyspnea and bleeding.

Pleural Effusions, Pneumothorax, and Hemothorax

Many infectious and noninfectious diseases can lead to the accumulation of fluid, air, or blood between the lung and chest wall—pleural effusion, pneumothorax, and hemothorax, respectively. These accumulations may be asymptomatic when they are small, but they can cause significant dyspnea as they enlarge. Decisions about how, and whether, to intervene depend on the child’s overall disease trajectory and the goals of care. If a child’s underlying disease is very advanced, excellent end-of-life care might lead a team toward conservative medical management of symptoms. On the other hand, if a child is doing relatively well, other options such as surgical intervention or radiation therapy might be appropriate.

Treatment choice should be aligned with the patient’s goals of care, and it should be fitting to the clinical realities of the patient’s condition. But deciding whether or not an intervention will offer meaningful benefit in these situations is sometimes difficult. For example, when a patient has a malignant pleural effusion in the context of progressive cancer, draining the effusion will not stop it from quickly reaccumulating. Placing a pigtail catheter might provide transient relief, but these catheters are small and they often become clogged with tumor cells. A larger chest tube might drain the effusion more reliably and for a longer period of time, but many patients experience pain at the chest tube site, and the presence of a chest tube can limit a patient’s mobility and options for location of treatment. These important aspects of medical and surgical interventions must be anticipated and weighed against the benefits that might be conferred.

Once an effusion is drained, pleurodesis might prevent or slow the reaccumulation. Pleurodesis is a procedure in which foreign materials are placed between the pleural surfaces of the chest wall and the lung to induce inflammation, allowing the two pleural surfaces to permanently adhere to each other. In theory this avoids recurrence of a pneumothorax or pleural effusion. In adult patients, pleurodesis is generally performed thoracoscopically, and talc is used as the material of choice to seal the pleural surfaces together.⁴⁰

A more recent innovation is the insertion of a tunneled catheter for control of malignant pleural effusion. These are increasingly being used for ambulatory adult patients and can often be inserted by a thoracic surgeon as an outpatient procedure.⁴¹ This procedure involves placing a tunneled catheter under the skin and subcutaneous tissue, through the thorax, and into the pleural collection of fluid. The exterior portion of the catheter can then be attached to a sterile collection bottle to perform serial drainages of the effusion as it reaccumulates, to maintain comfort. One study of 250 insertions in adults suggested that this approach is superior compared with talc pleurodesis, due primarily to the avoidance of a days-long hospital admission and long duration of result, usually until patients’ deaths.⁴² In pediatric palliative care, we often borrow from the experience accumulated in the world of adult medicine. This is certainly true in the case of pulmonary sources of distress. In all cases, the potential impact and benefit of the intervention must be weighed in light of the patient’s reality, including the trajectory of the patient’s disease and the overall goals of care.

Weakness in the Respiratory Musculature

Breathing difficulties can occur as a result of weakness in the respiratory musculature. Two inherited disease processes that cause severe muscle weakness are spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD).

SMA is a group of severe, genetic neuromuscular diseases that affect the spinal motor neurons. It has an estimated incidence of 1 in every 6,000 to 1 in 10,000 live births.⁴³ It is caused by a loss or defect of the SMN1 gene. The SMN1 gene is responsible for providing instruction to make the SMN protein, which in turn maintains healthy motor neuron function. With the degeneration of the spinal motor neurons, there is progressive muscle atrophy, weakness, and paralysis.⁴⁴ Included in the atrophy and weakness are the intercostal muscles, resulting in an irregularly bell-shaped chest and an abnormal breathing pattern. Type-1 and Type-2 SMA have the greatest impact on the respiratory system.

Type-1 SMA is the most common (45% of all cases) and most severe; the onset in children has been noted to be between birth and 6 months.⁴⁵ Respiratory insufficiency is typically seen at birth and issues may even have been noted prior to birth with reported decreased intrauterine movement (Type-1). Due to the weakness of the respiratory system, a caregiver or clinician may note a weak cry, an irregularly bell-shaped chest, and irregular breathing. The chest wall takes on a bell-shaped appearance resulting from poor expansion of the ribcage. Because of respiratory muscle weakness, the infant is typically seen “belly-breathing” (paradoxical breathing).^45,46 With this type of breathing, the abdomen protrudes and the chest wall flattens on inspiration instead of the chest wall expanding.^43,46 In this case, the infant is utilizing accessory muscles to generate each breath. This pattern of breathing is not effective for long periods of time and can result in respiratory fatigue or failure. Once the respiratory system has reached this point, adjunct therapies may be needed to support the child’s breathing. These therapies may include supplemental oxygen or NIV such as bilevel positive airway pressure (BIPAP) or continuous positive airway pressure (CPAP). Children with SMA may also have difficulty swallowing food as well as managing oral secretions due to bulbar (cranial nerves 9–12) dysfunction.⁴³ Dysphagia may also impact the respiratory system due to aspiration, leading to risk of pneumonia, and can affect nutrition. Providers and families will need to think about feeding tube options for their child to provide long-term nutritional support.

Scoliosis or curvature of the spine is believed to occur with an incidence of 60–90% within the Type-1 and -2 SMA. This can contribute to respiratory compromise since the chest wall is restricted and cannot move in an effective manner and proper lung expansion is inhibited.⁴⁴ This, in turn, can lead to areas within the lungs becoming atelectatic. Adjunct therapies may need to be utilized to support the child’s breathing. The child, secondary to poor chest movement and a weak cough, may not mobilize pulmonary secretions independently. Assisted cough and postural drainage therapies should be considered part of a proactive care approach. Postural drainage entails placing the child in positions that will encourage secretions to mobilize into the larger airways for removal. Cough assist is a cycle of applied positive and then negative pressure. The positive pressure will assist the child in taking deeper breaths where the negative pressure acts as an artificial cough in order to help remove pulmonary secretions. The strength and function of the child’s respiratory system/musculature should also be routinely assessed by pulmonary function testing (PFT), which measures lung volumes, airflow, and gas exchange. A decline or drop in PFT results may reflect a weakening of the respiratory system. When the respiratory system/musculature weakens to a point where the child is having nocturnal hypoxemia or hypoventilation, NIV may be initiated. This may avoid or prolong the need for advanced ventilation strategies, including the need for a tracheostomy.⁴³

New therapeutic approaches may have a positive effect not only on survival, but also on respiratory status. Nusinersen has become commercially available after two successful double-blind clinical trials in early- and late-onset SMA.⁴⁷ There is evidence that infants with SMA1 may have improved ventilation-free survival at 24 months compared to a historic cohort of patients.^48,49

DMD is an inherited X-linked genetic disorder with an estimated incidence of 1 in every 3,600–6,000 live male births.⁵⁰ With this disease process, the gene codes for the protein dystrophin are deleted or mutated. Dystrophin is made within all muscle cells of the heart and skeletal muscle fibers. It functions to both support and protect muscle fibers. If dystrophin is missing or defective, as in DMD, muscle cells can become damaged and do not work efficiently. Damaged cells will lead to an inflammatory process, which causes even further muscle damage. DMD is more commonly seen in boys, although in rare cases it can affect girls although it is usually milder.⁵¹

Muscle weakness/atrophy significantly impacts the cardiopulmonary system. Typically, by the child’s early teens, respiratory and heart muscles have begun to be altered by the diseases’ progression. Cardiac impairments present as cardiomyopathy or cardiac arrhythmias,^52,53 which can impact circulation and thus affect all of the body’s tissues and organs that depend on optimal cardiopulmonary function. The respiratory musculature, inclusive of the diaphragm, will also become progressively weaker. This in turn will influence how well the child’s chest expands and their ability to cough and clear pulmonary secretions. PFT should be routinely performed to monitor for early signs of respiratory compromise.⁵¹

Many of these young patients will also have skeletal problems secondary to progressive muscle degeneration and weakness, and postural compensation and contractures/deformities are likely to occur, including scoliosis.⁵³ This in turn will create respiratory issues since the chest is not able to move optimally. As the individuals’ disease progresses, the inability to create a strong cough and take deep enough breaths can lead to other pulmonary complications including atelectasis, retained pulmonary secretions, hypoventilation, and respiratory failure. Postural drainage and cough assist may help to remove pulmonary secretions. If hypoxemia or hypoventilation occur, NIV may be necessary. If a child’s respiratory status worsens to the point of respiratory failure in the context of either disease progression or pulmonary infection, mechanical ventilation may be necessary as a temporarily or long-term solution.

There is no known cure for either disease. However, as medical treatments have advanced, life expectancy has increased into young adulthood. Caregivers may have an emotional journey watching their child with a debilitating disease, and the complications that can occur can be overwhelming. As the function of the respiratory systems declines, there should be ongoing discussions between the interdisciplinary team, the caregiver(s), and the child/young adult regarding immediate and long-term goals with respect to quality of life and acceptable forms of intervention.

NIV includes face mask, nasal mask, Oxy-Hood, high flow nasal cannula (HFNC), CPAP, and BIPAP. HFNC, CPAP, and BIPAP are more commonly seen in children, thus discussion will focus on these. There are indications for use of NIV in infants and children with both acute and chronic conditions. The use of NIV is growing. In infants, NIV can be used for alveolar recruitment, post-extubation, hypotonia with respiratory insufficiency, conditions associated with a loss of lung volume, and obstructive airway disease (e.g., tracheomalacia, bronchomalacia, and laryngomalacia). NIV is indicated in children for respiratory insufficiency associated with acute lung injury, pneumonia, asthma, bronchitis, pulmonary edema associated with renal or cardiac failure, cystic fibrosis, neuromuscular disease, neurologic disorders associated with respiratory depression, chest wall deformities, post-extubation, and obstructive sleep apnea.^54,55,56 These examples represent only a few indications. Noninvasive positive pressure ventilation (NIPPV) is used in children with chronic hypoventilation secondary to muscular weakness as seen in muscular dystrophy and spinal muscular atrophy.⁵⁷ There are contraindications to noninvasive respiratory support, including if the child had previous unsuccessful NIV or CPAP, frequent and severe apneas, tracheoesophageal fistula, craniofacial abnormalities that may prevent application, untreated pneumothorax, or neuromuscular disorders associated with severe respiratory depression. NIV support is not effective in children with moderate to severe acute respiratory distress (ARDS) with respiratory failure, untreated pneumothorax, facial injuries, traumatic injuries, burns, risk of aspiration, severe hemodynamic compromise, patients undergoing upper gastrointestinal tract surgeries, or those with increased intracranial pressure.⁵⁸ While there are interventions that can provide life-sustaining care, this care may not be appropriate for every family.

HFNC is a form of NIV. It delivers a mixture of air and oxygen via a plastic tubing and sits inside the nostrils.⁵⁹ The definition of high flow varies with the age of the patient and is not universal. Flows greater than or equal to 1 liter per minute (LPM) is considered high flow in neonatal patients, but flow ranges for older children are not well established in literature. Flows greater than or equal to 3 LPM meet criteria for high flow in toddlers, and flows greater than or equal to 5 LPM is considered high flow in older children. The assumed benefits of HFNC are realized from maximum mucociliary clearance, reduced inflammatory reactions, inhibition of the bronchoconstrictor reflex, reduced airway resistance, and a variable degree of washout of the nasopharyngeal dead space, but the evidence for positive pressure delivery with HFNC is mixed and highly variable.⁵⁸ All forms of noninvasive respiratory support require adequate humidification to prevent drying of the mucus membranes.

Among the greatest challenges with use of NIV in infants and children are the interfaces and delivery devices. Patient interfaces and headgear have expanded and include a wider variety of total face masks, nasal masks, nasal pillows, and, most recently, cloth masks.⁵⁸ Clinicians should be mindful that despite improved masks patients may not tolerate the excessive flow associated with NIV. It may be necessary to consider both nonpharmacological and pharmacological interventions for anxiety management which could help to improve adherence.⁵⁷ The facial areas will need close monitoring for skin breakdown and pressure ulcers. A pulse oximeter and a cardiac monitor are recommended for all forms of oxygen. Close monitoring of oxygen saturations will be needed, but this may change as the child nears death. Transition toward end of life may indicate another discussion point where clinician and family will need to decide if NIV is helpful or if it is causing discomfort. Loving families may decide to continue this intervention while others may decide to stop it.

Recent evidence advocating for less invasive ventilation has been a catalyst to increase the study and use of CPAP in infants and pediatric patients. CPAP refers to the administration of continuous positive airway pressure during all phases of the respiratory cycle of a spontaneously breathing patient. CPAP is used in patients with hypoxemia related to alveolar collapse or symptoms of obstructive sleep apnea or upper airway collapse.⁵⁷ In the case of a patient with a viral illness in which the patient needs a limited period for recovery; CPAP or another source of NIV may be appropriate, although not without discomfort from a tight-fitting mask that may be difficult to wear if patient has claustrophobia. The goal of CPAP is to restore adequate functional residual capacity to correct, reverse, or minimize alveolar collapse and reduce the work of breathing because it unloads the work of the inspiratory muscles and allows effective inflation in excess of the opening pressures of the lungs.⁵⁸ The level of CPAP required to accomplish this goal will vary depending on the child and the disease process. Infants typically require 5–8 cm of water (cm H₂O), but disorders associated with low lung compliance may require CPAP levels in excess of 10 cm H₂O.⁵⁷ Pressure-constant delivery devices are preferred to provide constant pressure and prevent alveolar collapse. When adequate CPAP levels are achieved, the work of breathing improves, and oxygen saturations increase. Weaning from CPAP should start once the disease process is identified and improving. CPAP can also be used nocturnally for the treatment of obstructive sleep apnea.^56,58 Weaning from CPAP typically involves transition to HFNC or nasal cannula.

BIPAP is a form of noninvasive positive pressure ventilation that allows unrestricted spontaneous breathing at any moment of the ventilatory cycle. Depending on spontaneous breathing activity, it can be subdivided into no spontaneous breathing continuous (CMV)-BIPAP or spontaneous breathing at a lower pressure level intermittent (IMV)-BIPAP.⁶⁰ It is often used in the treatment of lung disease and respiratory weakness. It provides both inspiratory and expiratory pressure. It is commonly used in pediatrics for the treatment of impending respiratory failure, acute or chronic respiratory insufficiency secondary to pulmonary disease, neuromuscular disease, airway obstruction, infectious process, or post-extubation management to avoid intubation or reintubation.⁵⁴

Over the past 20 years the use of NIV has led to a 5- to 15-fold increase in the use of mechanical ventilation worldwide. The worldwide prevalence ranges from 2.1 to 13.7/100,000 in children.^{61,62,63,64,65,66,67,68,69} NIV is considered the standard of care for a range of medical conditions which lead to sleep-disordered breathing and chronic respiratory insufficiency or failure, with a greater proportion of those starting on home mechanical ventilation surviving to adulthood.^62,70

Many children who require NIV are medically complex. This includes children with chronic conditions that affect multiple body systems or progressive conditions associated with deteriorating health with limitations on life expectancy and dependence on technology.⁷¹ Caregiving for this cohort of children is indeed challenging. Caregivers often reported sleep deprivation and financial and social hardships.⁷²

Invasive Ventilation

Intubation

If NIV becomes ineffective or intolerable, then the next step is endotracheal intubation. Intubation can provide stability for the patient who is experiencing respiratory distress and offer recovery while other interventions are used to maximize respiratory status.⁷³ This intervention requires thoughtful consideration and discussion as endotracheal intubation and mechanical ventilation can be damaging to the lungs. Will this intubation allow time to rest and heal, for the child to return to baseline? Intubation can also make interaction and meaningful time with family difficult because of the need for sedation to allow tolerance of the endotracheal tube. Frequent conversations should occur between the medical team and family. The patient’s and family’s goals should be at the forefront of conversations. In the case of progressive disease, it is likely that even if the child recovers from this acute event they may need intubation again in the future. Therefore, it is an important conversation to have with the child’s family. If intubation were to occur again and no recoverable condition is found, it may be reasonable to discuss a time-limited trial on the ventilator. Endotracheal intubation is not meant to be a long-term solution but it can be a bridge to another intervention like a tracheostomy. It will be important to discuss all options with the caregiver/family.⁷³

Tracheostomy

The past decade has seen an increase in the number of tracheostomies performed in children. Children with tracheotomies represent a very complex cohort of patients with sustained reliance on the tracheostomy and related medical technology for long-term survival. While it is commonly performed in the adult population, it is less common in pediatrics because no consensus exists determining how long a child should remain endotracheally intubated.⁷⁴

Tracheostomies have been increasingly performed in children with complex and chronic conditions, for management of upper airway obstruction, prolonged ventilation, abnormal ventilatory drive, and irreversible neuromuscular conditions.^{75,76,77,78,79} More than 50% of children who receive a tracheostomy are under the age of 1 at the time of placement.⁸⁰ Decannulation rates in this population of children range from 28% to 51%, with the average time for tracheostomy remaining in place for 2 years.^{81,82,83,84,85,86,87} Tracheostomy is performed more frequently in children with chronic conditions including those with neurological impairment, congenital heart and lung disease, upper airway anomalies (either congenital or acquired secondary to prolonged intubation), or with a need for prolonged ventilation secondary to respiratory failure.⁷⁶ While tracheostomies provide lifesaving options for some children, it is not without difficulties. Once the child progresses past the possible complications of pneumothorax, pneumomediastinum, hemorrhage, tissue injury, and pulmonary edema, accidental decannulation and mucus plugging can remain ongoing challenges for the child and family.⁷⁴ For children who have chronic ventilator dependence who are preparing for discharge from acute care to the community, challenges exist as there are no clinical practice guidelines available to healthcare professionals to ensure a safe hospital discharge and community management.⁸⁸ Despite the mandate for national action by the Institute of Medicine in 2003 for children with special healthcare needs, there are no evidence-based guidelines for this complex subgroup of technology-dependent children.⁸⁹ Current recommendations for hospital discharge include an awake, trained caregiver present at all times and at least two trained family caregivers specifically for the child’s care because a single caregiver will rarely be able to shoulder the burden of care. Standardized equipment for monitoring, emergency preparedness, and airway clearance also must be a part of the discharge plan. These children often have several specialists involved in their care and benefit from having a medical home. The American Thoracic Society recommends a comprehensive medical home co-managed by the generalist and respiratory subspecialist. While care for this group of children may seem challenging for practitioners, families and patients see the benefits of care in the home setting.⁸⁸ Although care at home can be beneficial, it also has many challenges. Children often qualify for home health nursing, which allows families to work, care for other children, and allow time to coordinate care for the child. However, availability of nursing care can be limited, especially in rural communities. This shortage often leaves families providing around the clock care themselves, which leads to fatigue and financial hardship (Figure 24.1).

Related posts:

Quality in Pediatric Palliative Care Program Development The Language of Pediatric Palliative Care Practical Aspects of Palliative Care Communication Advanced Heart Disease Children’s Voices: The Experience of Patients and Their Siblings

Stay updated, free articles. Join our Telegram channel

Join