2004 ATS Consensus
Guidelines for Duchenne
Respiratory Management:
Still much left to do.
Jonathan D. Finder, MD
Associate Professor of Pediatrics
University of Pittsburgh School of Medicine
Clinical Director, Pediatric Pulmonology
Children’s Hospital of Pittsburgh
Background
• What prompted this project?
• To understand respiratory mgmt of
NMD, let’s go WAY back…
1950’s: Early respiratory care
experience in Polio shapes later
care in MD
1952 Emerson infant “iron lung”
End of the last century…
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•
•
•
•
Therapeutic nihilism abounds and yet…
New therapies and approaches available
Parental advocacy movement (PPMD)
Increasing scrutiny by managed health
DMD: an orphan disease, lacking
evidence, controlled trials
• The rise of the “guidelines movement”
Background: 1990’s
• 1994: PPMD founded
• 1994: In-exsufflator (Bach/Emerson)
• 1997: Bach’s prevention of pulmonary
morbidity in MD paper
• 2001: MD Care Act
2001
MD CARE Act:
Muscular
Dystrophy
Community
Assistance
Research and
Eductaion
The Chasm
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•
•
•
MD is relatively rare (1:3,000 boys)
Few large controlled studies
The medical literature is largely “anecdotal”
Withholding effective treatment to study it is
unethical (no clinical equipoise)
• CHASM between what is out there and what
patients receive
2001-2004 ATS Consensus
Project on Respiratory Care
• “A perfect storm”
– Availability vs accessibility of care
– Varying levels expertise/experience/comfort
among clinicians with newer therapies
– Lack of any published guidelines for MD care
– Parents demanding better care
ATS 2004 Statement
• ANTICIPATORY approach to respiratory care
• NON-INVASIVE management emphasized
• COLLABORATIVE care emphasized:
–
–
–
–
–
Pulmonologist
Nutritionist
Cardiologist
Orthopedist, Physiatry
Physical, speech, and occupational therapists;
psychiatry, pastoral care as needed
Gaining World-wide Acceptance…
Italian translation courtesy of PPMD Italy
Guidelines Continue to Emerge
• 2005: Corticosteroid treatment in DMD:
– Moxley, et al. Neurology 2005 Jan 11;64(1):13-20
• 2007: SMA Care guidelines:
– Wang, et al. Consensus Statement for Standard of
Care in Spinal Muscular Atrophy. J Child Neurol;
22; 1027, 2007
• 2007: Perioperative management guidelines
in DMD:
– Birnkrant, et al. Chest. 2007; 132:1977-1986
• 2008? CDC/MD Care Consideration
Consensus projects continue…
And YET -- We are not done.
• EXAMPLE: Mechanical insufflation-exsufflation
– Physiologically proven to improve cough flows in
weak patients1
– Demonstrated efficacious in largely retrospective
analyses2
– Moderately expensive/yet cost effective
– In 2001 largely denied by payers
• This is what prompted initiation of ATS Project!
1. Chest 1993;104:1553-1562
2. Am J Phys Med Rehabil 2002;81:411-415
ATS respiratory guidelines
and MI-E
• 2001 - 2004, Finder and colleagues
crafted a respiratory guidelines
statement with support of PPMD, ATS,
and MDA
• Little “evidence;” much anecdotal data
• Strongly supports MI-E by unanimous
consensus
Insurance coverage of MI-E
• 2001: <10% all payers cover
• 2008: 65% of private payers do not
cover it for DMD
• 2008: majority of Medicaid payers DO
cover MI-E today
Even with Children’s of Arkansas, directly
across from the State House, MI-E does
not have Medicaid coverage in AK…
CoughAssist and Medicaid
State by State…
YES
NO
AL, AK, AZ, CA, CO,
CT, DE, ID, IL, IN, IA,
KY, ME, MD, MA, MI,
MN, MO, MT, NE, NH,
NJ, NM, NV, NY, NC,
ND, OH, OK, PA, RI,
SD, TN, TX, UT, VT, WI,
WY
AR, FL, GA, KS, LA,
MS, OR, SC, VA, WA,
WV
DC
(HI -?)
Expertise vs Experience
• Without sufficient training, prescribing
lags
• No data on use of MI-E in MD nationally
– MDA lacks central data repository like CFF
• MI-E in use in 49% of spinal cord injury
care centers despite efficacy in this pop
– J Spinal Cord Med. 2007; 30(2): 127–130.
Back to square one:
• Many unanswered questions
– Orthopedic (when to operate, and how)
– Cardiac care (cardioprotective approach)
• MORE advocacy needed
• More guidelines work (CDC) needed
• More research needed
The future
• A single standard of care
• Better training
• Centers of clinical excellence in MD
care (Cincinnati, Pittsburgh, eg)
• Greater advocacy from PPMD/MDA to
get insurance companies to join the
21st century
Patrick, age 26, graduating from Pitt Law
Thank you!