Browsing by Author "Lantos, John"

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    Recommendations for the design of therapeutic trials for neonatal seizures
    (Springer Nature, 2019-06) Soul, Janet S.; Pressler, Ronit; Allen, Marilee; Boylan, Geraldine; Rabe, Heike; Portman, Ron; Hardy, Pollyanna; Zohar, Sarah; Romero, Klaus; Tseng, Brian; Bhatt-Mehta, Varsha; Hahn, Cecil; Denne, Scott; Auvin, Stephane; Vinks, Alexander; Lantos, John; Marlow, Neil; Davis, Jonathan M.; Pediatrics, School of Medicine
    Although seizures have a higher incidence in neonates than any other age group and are associated with significant mortality and neurodevelopmental disability, treatment is largely guided by physician preference and tradition, due to a lack of data from well-designed clinical trials. There is increasing interest in conducting trials of novel drugs to treat neonatal seizures, but the unique characteristics of this disorder and patient population require special consideration with regard to trial design. The Critical Path Institute formed a global working group of experts and key stakeholders from academia, the pharmaceutical industry, regulatory agencies, neonatal nurse associations, and patient advocacy groups to develop consensus recommendations for design of clinical trials to treat neonatal seizures. The broad expertise and perspectives of this group were invaluable in developing recommendations addressing: (1) use of neonate-specific adaptive trial designs, (2) inclusion/exclusion criteria, (3) stratification and randomization, (4) statistical analysis, (5) safety monitoring, and (6) definitions of important outcomes. The guidelines are based on available literature and expert consensus, pharmacokinetic analyses, ethical considerations, and parental concerns. These recommendations will ultimately facilitate development of a Master Protocol and design of efficient and successful drug trials to improve the treatment and outcome for this highly vulnerable population.
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    When Adolescent and Parents Disagree on Medical Plan, Who Gets to Decide?
    (AAP, 2019-08) Hopkins, Kali A.; Ott, Mary A.; Salih, Zeynep; Bosslet, Gabriel T.; Lantos, John; Pediatrics, School of Medicine
    Duchenne muscular dystrophy (DMD) is an X-linked autosomal recessive disease affecting 16 to 20 per 100 000 live births.1,2 It is characterized by progressive muscle weakness due to a defect in the dystrophin gene. It typically leads to loss of ambulation by age 8 to 14 years,1 followed by cardiomyopathy and respiratory failure. Historically, adolescents with DMD have died at ∼20 years of age.1–3 As respiratory compromise occurs, patients are supported with noninvasive ventilation (eg, nasal bilevel positive airway pressure).3–6 When this becomes unsuccessful, patients may be candidates for tracheostomy; this often happens in the second or third decade of life.7 The decision of whether to proceed with tracheostomy is complicated and is most often left to the patient and family. Family members do not always agree. We present a case in which acute illness forced a minor and his family to face this decision earlier than is typical. The adolescent desired a tracheostomy to extend his life. The parents did not believe that a tracheostomy was in his best interest and felt that comfort care was the most appropriate approach. Experts comment on the ethical issues raised by medical decision-making in cases involving adolescents and life-and-death decisions.