Hello to my Pedi Endo family. Now that we are in summer, I am hoping that you have some “me time.” You might be traveling for the first time in a while.
Behind the scenes, the Board and Committees/SIGs are working on many projects, such as organizing the PES Annual Meeting (May 5-8, 2023, in always sunny San Diego). I thank the many people who filled out the survey on what they would like to see at the next PES Annual Meeting.
We are committed to many important social issues as shown in our Advocacy Corner. I suggest in your spare time, to peruse the website and check out the names of the Committees and SIGs to see what is their areas of current focus.
As a clinician, I am interested in helping to make our jobs more pleasurable, efficient, and with proper reimbursement. I am hoping we all share tips that can help your pedi endo brothers and sisters. (please email your tips to firstname.lastname@example.org with “PES Tips” as the subject and they will be reviewed by the Board and me). Do not think your tip is too small to share. My hope is to showcase these tips (without your name attached). These can be tips for those in private practice or to those working at an academic center.
I want to keep the newsletter informative yet also entertaining. Of course, some of you may want to skip all the information and go right to the fun parts, and other skip the entertaining parts and are looking for “data.”
So, I offer you a choice as I present two short cases:
Choose A for the Entertaining Case titled “Get your Facts in Order”, or B for the more informative case “Tips on Hashing out the Pituitary Disease”, (or both if you are all caught up on your grant writing and EMR messages).
A: Get Your Facts in Order
A mother brings in a 10-year-old boy with concerns of short stature. She proves to be the best historian I have seen. She relays to me heights and timing of puberty of generations in her family. I could not type fast enough. The endocrinologist (me) is drooling at the precision of information until she asks, “Why do you need all this information about puberty?” I respond, as you all would, “Oh, not only do heights run in the family, but so does the timing of growth and puberty”. She responds, “Yes, but that is not my kid”. (She was the neighbor) Moral, “Get Your Facts in Order” by first asking who is the adult.
B. Tips on “Hashing out the Pituitary Disease”
Neurosurgery sees a 13-year-old who has headaches and a pituitary mass (2x2x2 cm). They obtain the following lab studies and send you a referral for a new “prolactinoma”.
The laboratory studies showed:
Total T4 1.5 ug/dL (4.7-10), T3RU 28%
GH random 0.49 ng/ml
Prolactin 106 ng/ml (2-14)
Analysis: The prolactin is elevated though perhaps not as high as one might expect with a mass that size. Why was there a random GH level and not IGF-1? (Remember who ordered the tests). There is certainly a low T4. Could this be severe central hypothyroidism causing the high prolactin (TRH stimulates prolactin release, as well known to those who ever performed the good old TRH provocative test). Missing though was a TSH. Result: 859 uIU/ml (and later, thyroid antibodies +++). This case is a reminder that a TSH must be ordered even when the mass is in the pituitary and the T4 is low. That is my tip as you Hash(imoto) out the diagnosis.
So, here is my first tip on efficiency: Avoid rescheduling patients by planning ahead.
The Annual meeting is May 5-8, 2023. Plan to block out the day before as a travel day. We have committee and other ancillary meetings starting early on the 5th.
If you are considering attending the Board Review Course, you should plan to arrive early for a May 2-4 course.
I look forward to working with you, the Board, Degnon and all those working on projects for the PES.
Craig A. Alter, M.D.
New Meds and Tech from PES Drugs and Therapeutics Committee:
Anna Ryabets-Lienhard, DO, Christine Yu, MD, Emily Breidbart, MD
In August 2021, FDA approved SKYTROFA®(lonapegsomatropin-tcgd), the first pediatric long-acting growth hormone (GH). Lonapegsomatropin-tcgd is a human growth hormone (GH) that utilizes TransCon® technology allowing once weekly subcutaneous administration of GH. SKYTROFA®is indicated for the treatment of pediatric patients one year and older with GH deficiency and weights of at least 11.5 kg. The recommended subcutaneous dose is 0.24 mg/kg/week administered into the abdomen, buttocks, or thigh with a regular rotation schedule of the injection sites (FDA). It is available as a powder in single-dose, dual-chamber, prefilled cartridges at the doses of 3 mg, 3.6 mg, 4.3 mg, 5.2 mg, 6.3 mg, 7.6 mg, 9.1 mg, 11 mg, and 13.3 mg and delivered via an auto-injector (FDA).
Anna Ryabets-Lienhard, DO
On November 19, 2021, the FDA approved VOXZOGOTM (vosoritide) for the treatment of children over five years of age with achondroplasia and open epiphyses to increase linear growth. VOXZOGOTM was approved under an accelerated program based on improved annualized growth velocity (AGV); continued approval is contingent upon continued review and verification of clinical benefit in ongoing extension clinical trials. Vosoritide is a biological analog of C-type natriuretic peptide (CNP), which stimulates cartilaginous bone growth via endochondral ossification. The approved dose is approximately 15 mcg/kg administered subcutaneously once daily; a weight-based dosing chart is available in the FDA published prescribing information
(https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/214938s000lbl.pdf). VOXZOGOTM is provided as a lyophilized powder in a single-dose vial at 0.4 mg, 0.56 mg, or 1.2 mg. It requires reconstitution with a prefilled co-packed diluent (sterile water) delivered in a syringe prior to administration. Due to transient decrease in blood pressure that has been observed with VOXZOGO,TM the patient needs to be well fed and hydrated (approximately 240-300 mL of fluid) an hour prior to administration of the medication to reduce the risk of hypotension.
Virtual Symposium: Addressing Treatment Fatigue: Long-Acting Growth Hormone for the Pediatric Patient with GH Deficiency
Wednesday, July 13, 2022, 7:00 PM – 8:00 PM Eastern
- Interactive, Case-Based Learning
- Complimentary Personalized Online Poster Portal
- Whiteboard Animations
FACULTY: Andrew Dauber, MD MMSc, and Bradley S. Miller, MD, PhD
This virtual symposium, sponsored by Novo Nordisk, Inc., will address the latest advancements for childhood growth hormone deficiency (CGHD), a condition where the pituitary gland fails to produce enough growth hormone during childhood. Expert pediatric endocrinologists will discuss the challenges of current daily hormone therapies and will review important information regarding longer acting hormone formulations for the chronic management of CGHD.
New Steroids, New Treatments, and New Approaches to Congenital Adrenal Hyperplasia Webinar
Monday, July 18 from 4:00-5:00 PM EDT
Presented by Dr. Richard Auchus
Calling all regions! Are you planning an in-person meeting before the end of the year? Are you interested in a grant to invite a speaker? If you answered yes to these questions…please know the PES Board has allocated $2000 per meeting with plans to fund up to four Pediatric Endocrinology Regional Meetings per year. The intent is to support meetings of established Pediatric Endocrine groups, with a focus on regional involvement of multiple institutions and opportunity for Fellows to participate. The funds are provided to defray expenses of an invited speaker.
We are accepting grant applications for the fiscal year January 2022. Grant applications will be reviewed on a first-come basis and funds are limited. Please find the application here: Regional Grant – Pediatric Endocrine Society (pedsendo.org)
NEW! 2022 Obesity Exam Now Available:
This activity contains approximately 60 items that focus on a stepwise approach to the diagnosis and management of pediatric obesity and its comorbidities including a review of evidenced-based clinical practice. This activity is eligible for both MOC points and CME credits. Click here for more information and to register: PES Obesity SIG MOC/CME Activity – Pediatric Endocrine Society (pedsendo.org)
June Public Policy Spotlight:
Fellow Spotlight: Janet Lucien, DO
History Tidbit: H. Franklin Bunn (July 7, 1935- ) Submitted by Alan D. Rogol, MD, PhD
From the NIH:
Although the trend toward reduction of the NIH funding gap is gratifying, significant work remains before the NIH realizes a truly diverse scientific workforce representative of our nation. Research Project Grant Funding Rates and Principal Investigator Race and Ethnicity – NIH Extramural Nexus