Therapeutic drifts in neonatology - a cautionary tale

Sunday, April 27, 2025

2:00pm – 3:30pm HST

Location: Convention Center: 316 BC

CE: 1.5

Selected By: Neonatal

Chair(s)

Richard Martin, md

Professor
Case Western Reserve University School of Medicine
Cleveland, Ohio, United States
Julee Oei, MBBS FRACP MD (she/her/hers)

PROFESSOR
Newborn Care
Royal Hospital for Women
Randwick, New South Wales, Australia

Disclosure(s):

Richard Martin, md: I do not have any relevant financial / non-financial relationships with any proprietary interests.

Julee Oei, MBBS FRACP MD: Mallinkrodt (Ongoing) (Products/Services: Consultant, Contractor including contracted research, Contractor including contracted research, Research Support including clinical trials & the principal or named investigator, Speaker Honorarium including speakers bureau, symposia, and expert witness, Speaker Honorarium including speakers bureau, symposia, and expert witness)

Many important interventions in neonatology have evolved from ground-breaking randomised controlled trials (RCTs) that were conducted years ago in very different populations of infants to those managed in neonatal intensive care units (NICU) today. Many of these interventions have allowed increasingly sicker and smaller infants to survive, but how the interventions themselves are used have changed considerably from their original intent. In addition, the use of these interventions have led to entrenched practice and the development of staunch and immutable mindsets that have precluded further evaluation and research of the benefit, effectiveness, safety and acceptability of these practices in contemporary practice.

We explore four areas of interest that represent significant therapeutic drift in neonatal care (steroids, nitric oxide, therapeutic hypothermia, and donor human milk) and that are now used well beyond the original intent of the randomized trials that introduced these therapies including for increasingly premature infants.

1. Introduction - What is Therapeutic Drift and the role of randomised controlled trials in neonatal practice - Richard Martin
2. Steroids - walking down the Primrose Path for steroids- Rita Ryan
3. Nitric oxide - following the Yellow Brick Road for preterm babies with respiratory failure - Ju Lee Oei
4. Cooling - To cool or not to cool, that is the question for mild HIE - Lina Chalak
5. Milk - Human donor milk as a panacea - can we change the dogma? - Josef Neu
6. Wrap up - Is therapeutic drift preventable or should it be prevented?

Learning Objectives:

Understand how and why practice derived from clinical trials and research has evolved and is now used to treat very different infants today
Understand the need to consider further research to evaluate how interventions derived from past research fit the needs of today's infant in the nicu
Acknowledge knowledge gaps in using these interventions for the participants' own practice and to evaluate their research and clinical strategies to bridge these gaps.

Presentations:

2:00pm – 2:06pm HST

Introduction. What Is Therapeutic Drift and the Role of Randomised Controlled Trials in Neonatal Practice?
Speaker: Richard Martin, md – Case Western Reserve University School of Medicine
2:06pm – 2:27pm HST

Following the Yellow Brick Road for Treating Preterm Babies with Inhaled Nitric Oxide
Speaker: Julee Oei, MBBS FRACP MD (she/her/hers) – Royal Hospital for Women
2:27pm – 2:48pm HST

Human Donor Milk as a Panacea - Can We Change the Dogma?
Speaker: Josef Neu, Doctor of Medicine (he/him/his) – University of Florida College of Medicine
2:48pm – 3:09pm HST

Walking down the Primrose Path for Steroids
Speaker: Rita M. Ryan, MD (she/her/hers) – Case Western Reserve University School of Medicine
3:09pm – 3:30pm HST

To Cool or Not to Cool, That Is the Question for Mild Hypoxic Ischemic Encephalopathy
Speaker: Lina Chalak, MD, MSCS – UTSW