Abstract Details

Title Transition to Adulthood in Duchenne/Becker Muscular Dystrophy

Topic Neuromuscular and Clinical Neurophysiology (EMG)

Presentation(s) P5 - Poster Session 5 (5:30 PM-6:30 PM)

Poster/Presentation
Number 12-016

Objective To identify psychosocial factors affecting the transition to adulthood in patients with Duchenne Muscular Dystrophy (DMD) or Becker Muscular Dystrophy (BMD).

Background As the current standard of care has extended the life expectancy for DMD/BMD patients, more data is needed to fully understand the transition from childhood to adulthood.

Design/Methods We performed a retrospective chart review on patients ≥ 15 years of age with a clinical diagnosis of DMD or BMD who received care from January 1, 2008 to 2018 at the University of Kansas and the University of Rochester Medical Centers. Eligible participants were identified by local neuromuscular lists, and electronic medical record review using DMD/BMD as search criteria. Data was abstracted on demographics, education, employment, community resources utilized, finances, relationships, and medical factors relating to the transition to adulthood. Data is presented as frequency or median.

Results Eighty-five charts were identified: patients ranged in age from 16 to 56 years with a median of 23.5 years, and covered a 4-state region (Kansas, Missouri, New York, and Texas) and Ontario, Canada. The majority were diagnosed with DMD (76.5%), who were on average 7 years younger than BMD. Over half (61%) were high school graduates or beyond: 5% Associate’s degrees; 9% Bachelor’s degrees; and 1% Master’s degrees. The most frequent fields of study were graphic design (16%) or computer science (13%). Twenty percent did not have their highest level of education documented. One third of patients stated some form of occupation, most frequently in education and administrative support (25%). The majority were still living with parents (82%), with 1 individual stating they were homeless. A minority reported having end of life discussions (21%).

Conclusions

The recording of the psychosocial elements underpinning the transition from childhood to adulthood is inconsistent or underreported. A prospective study will further elucidate this transition.

Authors/Disclosures
Jeffrey Statland, MD (University of Kansas Medical Center) PRESENTER	Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Arrowhead. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for ML Bio. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Epic Bio. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Armatus . Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Kate Therapeutics. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Vita Therapeutics. Dr. Statland has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Dyne Therapeutics. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Avidity . Dr. Statland has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Fulcrum Therapeutics. Dr. Statland has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Vertex . The institution of Dr. Statland has received research support from NIH. The institution of Dr. Statland has received research support from FSHD Society. The institution of Dr. Statland has received research support from Friends of FSH Research. The institution of Dr. Statland has received research support from FSHD Canada. The institution of Dr. Statland has received research support from MDA.
	No disclosure on file
Debra Guntrum, MS, FNP (University of Rochester Medical Center)	Ms. Guntrum has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Avexis. Ms. Guntrum has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sarepta. Ms. Guntrum has received personal compensation in the range of $500-$4,999 for serving as a Consultant for PTC.
Emma Ciafaloni, MD, FAAN (University of Rochester Medical Center)	Dr. Ciafaloni has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Argenx, Alexion, Sarepta, UCB, Hoffman-LaRoche, Biogen. Dr. Ciafaloni has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis, AnnJi Pharmaceutical, ML-BIO, Avidity. The institution of Dr. Ciafaloni has received research support from CDC, CureSMA, FDA, Orphazyme, Sarepta, PCORI, Neurogene. Dr. Ciafaloni has received publishing royalties from a publication relating to health care.

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