We present respiratory results from the Center for Disease Control and Prevention (CDC) multidisciplinary initiative to develop care considerations for patients with Duchenne muscular dystrophy.
The Respiratory Panel had 10 members representing pediatric and adult pulmonology, respiratory/physical therapy, neurology, rehabilitation medicine, sleep medicine and critical care. Using the Rand/UCLA Appropriateness Method (RAM), interventions and assessments were anonymously rated in three rounds using hypothetical clinical situations presented in a matrix format. An ordinal scale of 1–9 was used to categorize care as appropriate, inappropriate, or uncertain. Scenarios rated as “appropriate without disagreement” (n=115) were rated for necessity; then, the ratings were translated into recommendations.
Recommendations include: In the outpatient clinic, regularly assess forced vital capacity (FVC), oxyhemoglobin saturation by pulse oximetry (SpO2), peak cough flow (PCF), end-tidal carbon dioxide level (ETCO2) and (optionally) maximum inspiratory and expiratory pressures. Measure SpO2 at home to assess respiratory status during acute illnesses in patients with PCF < 270 lpm. Use home oximetry, oximetry-capnography, or polysomnography to assess gas exchange during sleep in patients with signs or symptoms of hypoventilation. Use these assessments to provide respiratory interventions before predictable complications arise. Initial interventions include lung volume recruitment for patients with FVC < 40% predicted. Add manual and mechanically assisted cough when the PCF is < 160–270 lpm; nocturnally assisted ventilation when the patient develops symptoms of hypoventilation; and daytime assisted ventilation, when symptoms occur during wakefulness despite nocturnal ventilation. Noninvasive ventilation was strongly recommended, but potential indications for tracheostomy were also identified. The statement also discusses the equipment and personnel needed for optimal respiratory care; recommendations for vaccines, antibiotics and anesthetics; and respiratory care at the time of surgery. Additional details will be presented at the meeting.
The RAM's structured quantitative methodology highlighted areas of consensus and disagreement, facilitating our development of recommendations for respiratory care of patients with DMD.
The CDC and other sponsors plan to disseminate the guidelines widely and to evaluate their efficacy.
David Birnkrant, Grant monies (from sources other than industry) Funded by the Center for Disease Control and Prevention and the National Center for Birth Defects; Grant monies (from industry related sources) Dr. Amin: study funding from Procter and Gamble and the Cystic Fibrosis Foundation Therapeutics Development Network and Vertes.; No Product/Research Disclosure Information