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6 Dec, 8 tweets, 4 min read
In discussing therapies for skeletal dysplasia, @mikebbober highlights these are a heterogeneous group of unique disorders #HotTopicsNeo2020 #neoEBM
Best-practice guidelines for delivery and evaluation of patients with skeletal dysplasia ajog.org/article/S0002-… and skeletaldysplasia.org
Trial for a new enzyme-replacement therapy (ENB-0040) for severe hypophosphatasia: nejm.org/doi/full/10.10…
Asfotase Alfa approved by @US_FDA in 2015 for treatment of pediatric onset hypophosphatasia
Goals of early care of infants with osteogenesis imperfecta
One year later ...
Take home points about caring for infants with #OsteogenesisImperfecta
Several available treatments and many emerging treatments in development for treatment of skeletal dysplasia ncbi.nlm.nih.gov/pmc/articles/P…

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More from @EBNEO

Evidence-Based Neo Profile picture
7 Dec
To conclude "New Approaches to Cooling", Sudhin Thayyil (@SudhinThayyil) of @imperialcollege discusses the “Hypothermia for Encephalopathy in Low and Middle-Income Countries (HELIX) Trial”

Trial protocol: pubmed.ncbi.nlm.nih.gov/28923118/

#HotTopicsNeo2020 @HotTopicsNeo #neoebm Image
Thayyil: current guidelines recommend therapeutic cooling in LMIC settings. Supported by MA findings, but based on small, low quality studies.

#HotTopicsNeo2020 @HotTopicsNeo #neoebm ImageImage
Thayyil: Aim, setting, eligibility, sample size and target outcomes for the HELIX trial.

#HotTopicsNeo2020 @HotTopicsNeo #neoebm ImageImageImageImage
Read 8 tweets
Evidence-Based Neo Profile picture
7 Dec
Next up in "New Approaches to Cooling" Deirdre Murray of @CUH_Cork discussing “Neurodevelopmental Outcomes after Mild HIE.” Read more about Prof. Murray and her work: infantcentre.ie/who-we-are-2/o…

#HotTopicsNeo2020 @HotTopicsNeo Image
Murray begins by defining the population discussed in her lecture: those with encephalopathy 2/2 perinatal asphyxia.

#HotTopicsNeo2020 @HotTopicsNeo Image
How do infants with different HIE severity grades perform on IQ testing at 5 years of age? Summary from:
pubmed.ncbi.nlm.nih.gov/27650049/, which includes additional outcomes measures.

Take home point: mild and moderate HIE infants similar.

#HotTopicsNeo2020 @HotTopicsNeo Image
Read 6 tweets
Evidence-Based Neo Profile picture
7 Dec
Last in the Rx for BPD trio at #HotTopicsNeo2020: Dr. Reese Clark of @MEDNAX with: "What Drugs are we Using? Practice Variability and Potential Harm." Read more about Dr. Clark's distinguished care:
blog.mednax.com/mednax-neonato…
@HotTopicsNeo Image
Reese: Talk to focus on steroid use. To begin, what postnatal steroids are we using? Dexamethasone and hydrocortisone most common systematic exposures, budenoside most common inhaled exposure.

#HotTopicsNeo2020 @HotTopicsNeo Image
The unstoppable pendulum of postnatal steroid use; data from @MEDNAX clinical data warehouse suggests use again on the rise.

#HotTopicsNeo2020 @HotTopicsNeo Image
Read 4 tweets
Evidence-Based Neo Profile picture
7 Dec
Next up in #HotTopicsNeo2020: Erik Jensen of @ChildrensPhila discussing how the baseline risk of BPD impacts the number needed to treat when considering effective therapies.

chop.edu/doctors/jensen…

@HotTopicsNeo Image
Presentation builds on recently published report in @JPediatr: drugs to Prevent Bronchopulmonary Dysplasia: Effect of Baseline Risk on the Number Needed to Treat
jpeds.com/article/S0022-…

#HotTopicsNeo2020
Jensen: objectives of talk:

#HotTopicsNeo2020 @HotTopicsNeo Image
Read 8 tweets
Evidence-Based Neo Profile picture
7 Dec
From the last session on periviability at @HotTopicsNeo, let’s get the pulse of the #neoEBM community with a few surveys (thread) #HotTopicsNeo2020
What is the earliest gestational age, assuming dating is correct, where you (or your hospital) routinely offer active postnatal resuscitation if desired by the family
If an infant at 22-24 weeks GA has evidence of inadequate organ perfusion (low urine output, decreasing BP), what is your first line treatment:
Read 6 tweets
Evidence-Based Neo Profile picture
7 Dec
First up in: "Pharmacotherapy for BPD: We Know Less than We Think We Do" at #HotTopicsNeo2020 is Dr. Gerri Baer (@thegerribaer), Team Leader for Neonatology @US_FDA, discussing "Challenges in Studying Drugs for BPD"
@HotTopicsNeo #neoEBM Image
Baer: no (zero, zilch, none!) drugs approved in the USA to prevent or treat BPD.

Highlights various cohorts demonstrating the ongoing high burden of BPD:

#HotTopicsNeo2020 @HotTopicsNeo Image
Baer: broad view of challenges to BPD Rx, illustrated by 3 buckets: understanding phenotypes/endotypes, definitions and classification and choosing adequate endpoints.
#HotTopicsNeo2020 @HotTopicsNeo Image
Read 5 tweets

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