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The Evolving Role of Current and Novel Therapies in Improving Outcomes in Adults with PKU

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The Evolving Role of Current and Novel Therapies in Improving Outcomes in Adults with PKU The Evolving Role of Current and Novel Therapies in Improving Outcomes in Adults With PKU The symposium is not part of the official ACMG Annual Meeting program, and ACMG does not approve or endorse any commercial products or services discussed during the symposium or offered for sale by the corporate supporter of the symposium. ACMG has reviewed and approved this symposium as appropriate for presentation as an independent educational activity held in conjunction with the ACMG Annual Meeting. Disclosures Barbara K. Burton, MD, has a financial interest/relationship or affiliation in the form of: Consultant for BioMarin Pharmaceutical Inc.; Horizon Pharma; REGENXBIO Inc.; Reneo Pharmaceuticals, Inc.; Shire; and Viking Therapeutics. Grant/Research Support from Sangamo Therapeutics, Inc.; Shire; and Ultragenyx Pharmaceutical. Speakers Bureau participant with Alexion Pharmaceuticals Inc.; Sanofi Genzyme; and Shire. Advisory Board for Alexion Pharmaceuticals Inc. and Shire. Other Financial or Material Support from Horizon Pharma for the Data and Safety Monitoring Board. BioMarin Pharmaceutical Inc. for clinical trial steering committee. Barbara K. Burton, MD, does intend to discuss either non–FDA-approved or investigational use for the following products/devices: treatments for phenylketonuria. This CME activity is jointly provided by Medical Learning Institute, Inc. and PVI, PeerView Institute for Medical Education. This activity is supported by an educational grant from BioMarin Pharmaceuticals Inc. Disclosures Nicola Longo, MD, PhD, has a financial interest/relationship or affiliation in the form of: Consultant for Aeglea BioTherapeutics; BioMarin; Censa Pharmaceuticals; Dimension Therapeutics; Genzyme Corporation/sanofi-aventis U.S. LLC; HemoShear Therapeutics, LLC; Horizon Pharma plc; Lumos Pharma; Mitobridge, Inc.; Moderna Therapeutics; Pfizer Inc.; Retrophin, Inc.; and Stealth BioTherapeutics Inc. Grant/Research Support from Lumos Pharma. Clinical Trial Support for Aeglea BioTherapeutics; BioMarin; Censa Pharmaceuticals; Genzyme Corporation/sanofi-aventis U.S. LLC; Horizon Pharma plc; Pfizer Inc.; Protalix Ltd.; Retrophin, Inc.; Shire; Stealth BioTherapeutics Inc.; and Ultragenyx Pharmaceutical. Travel Support for Cello Health and Sigma-Tau Pharmaceuticals, Inc./Alfasigma USA, Inc. Nicola Longo, MD, PhD, does intend to discuss either non–FDA-approved or investigational use for the following products/devices: treatments for phenylketonuria. This CME activity is jointly provided by Medical Learning Institute, Inc. and PVI, PeerView Institute for Medical Education. This activity is supported by an educational grant from BioMarin Pharmaceuticals Inc. Disclosures CME Reviewer Medical Director Staci M. Kallish, DO Kirk A. Tacka, PhD University of Pennsylvania School of Medicine PVI, PeerView Institute for Medical Education Philadelphia, Pennsylvania Kirk A. Tacka, PhD, has no financial Staci M. Kallish, DO, has no financial interests/relationships or affiliations in relation to interests/relationships or affiliations in relation to this activity. this activity. The associates of Medical Learning Institute, Inc., the accredited provider for this activity, and PVI, PeerView Institute for Medical Education do not have any financial relationships or relationships to products or devices with any commercial interest related to the content of this CME activity during the past 12 months. Visit us at www.peerview.com/PKU18 • Watch for the onDemand version in the coming weeks • Download the slides and infographic Practice Aids • Apply for CME credit • Join the conversation on Twitter @PeerView • Need more information? Send an email to [email protected] Download Infographic Practice Aids Introduction Barbara K. Burton, MD Professor of Pediatrics Northwestern University Feinberg School of Medicine Photo Director, PKU Clinic Pending Ann & Robert H. Lurie Children’s Hospital of Chicago Chicago, Illinois Phenylketonuria (PKU)1 Autosomal Recessive Inborn Error Characterized by Mutations in Gene of Metabolism Affecting Phenylalanine Encoding for the Hepatic Enzyme, (Phe) Catabolic Pathway Phenylalanine Hydroxylase Chromosome 12 PAH gene (12q22-12q24.2) >500 known mutations Identified by newborn screening Incidence: 1:10,000 to 1:15,000 1. Vockley J et al. Genet Med. 2014;16:188-200. Phenylalanine Metabolism: A Simplified View1 Phenylalanine Hydroxylase (PAH) Phenylalanine Tyrosine BH4 (cofactor) and O2 1. Vockley J et al. Genet Med. 2014;16:188-200. Alterations in Phenylalanine Metabolism in PKU1 PAH Defective BH (cofactor) and O Phenylalanine 4 2 Tyrosine Phenylpyruvic acid Phenyllactic acid Phenylacetic acid 1. Vockley J et al. Genet Med. 2014;16:188-200. Clinical Manifestations of Neurocognitive and Neuropsychiatric Effects in Early Treated PKU1-6 Executive Function Deficits Psychiatric Symptoms Psychological Behavioral • Depression • ADHD • Agoraphobia • Anxiety • Self-harm • Agitation • Mood swings • Schizophrenia Neurological Abnormalities • Spasticity • Gait disturbances • Tremor • Seizures 1. Channon S et al. Neuropsychology. 2004;18:613-620. 2. Brumm VL et al. Mol Genet. 2010;99:S59-S63. 3. Sullivan JE, Chang P. J Pediatr Psychol. 1999;24:281-299. 4. Burton BK et al. Mol Genet Metabol. 2013;108:8-12. 5. Waisbren SE, Levy HL. J Inherit Metab Dis. 1991;14:755-764. 6. Pietz K. Curr Opin Neurol. 1998;11:679-688. Executive Function Deficits in PKU1 P < .001 25 21 a 20 15 10 5 Range, % 5 Children in Severe Children 0 Control PKU a Based on Behavior Rating Inventory of Executive Function (BRIEF) Global Executive Composite; severe range is >1 SD above the mean. 1. Anderson VA et al. Child Neuropsychol. 2002;8:231-240. Neuropsychiatric Comorbidities in Adults With PKU1 25 General population a PKU 19.5 20 b Rates of intellectual disability, 15.6 anxiety, and depression 15 significantly higher for PKU 11.8 cohort vs general population 10 9.2 Adults, % Adults, a cohort across all age groups 4.8 (20-29, 30-39, 40-49, 50-59, 5 60-69, and 70+ years of age) 0.6 0 Intellectual Anxiety Depression Disability a P < .0001. b P = .0001. 1. Bilder D et al. Mol Genet Metabol. 2017;121:1-8. Overall Burden of PKU1 PKU Burden Health Status Psychological Family Life Social Function • Cognitive Function • Future family • Isolation function • Mood changes • Psychological • Relationships • Symptoms • Emotions impact on • School • Monitoring • Feelings family members • Work 1. Regnault A et al. Oprhanet J Rare Dis. 2015;10:59-76. Moving on to This Evening’s Agenda First, we’ll review current guidelines for PKU management and consider their relevance to adult patients Then, we’ll discuss available data on approved and emerging adjunctive pharmacologic approaches for the treatment of PKU Be prepared for polling questions both before and during tonight’s scientific sessions Contemporary Practice Guidelines for Maintaining Metabolic Control and Mitigating the Neurocognitive Impairments of PKU How Are They Pertinent to Adult Patients? Nicola Longo, MD, PhD Professor and Chief Medical Genetics/Pediatrics Photo University of Utah Pending Salt Lake City, Utah Goals of Treatment in Patients With PKU1 120 to 360 μM Control blood [Phe] in all patientsa Ongoing Monitoring • Phe and tyrosine testing • Nutritional markers and micronutrients • Plasma amino acids and protein status • Bone density • Neuropsychological testing aEuropean guidelines: recommended blood [Phe] range for patients > 12 years of age =120-600 μM2 1. Vockley J et al. Genet Med. 2014;16:188-200. 2. van Wegberg AMJ, et al. Orphanet J Rare Dis. 2017;12(1):162. Dietary Management of PKU1,2 • Omission of all high-protein food • Inclusion of modified low-protein food • Replacement of restricted protein and calories with metabolic formulas − Contain Phe-free proteins, fats, carbohydrates − Supplemented with tyrosine, vitamins, minerals However … 1. Vockley J et al. Genet Med. 2014;16:188-200. 2. Singh R et al. Genet Med. 2014;16:121-131. Dietary Management Also Contributes to Overall Burden of PKU1 Phe-Free Health Status Supplements • Cognitive PKU • Daily life function Burden • Social • Symptoms • Taste • Monitoring • Convenience Psychological Social Diet Family Life Function Function • Daily life • Future family • Mood • Isolation • Food • Impact on changes • Relationships • Social family • Emotions • School • Psychological members • Feelings • Work • Family Consequently… 1. Regnault A et al. Oprhanet J Rare Dis. 2015;10:59-76. Compliance With Dietary Treatment Decreases With Age1 Plasma [Phe] increases with age as children have a /L more varied diet and start mol μ having a social life. ], Phe [ 1. Viau KS et al. J Inherit Metab Dis. 2001;34:963-971. Compliance With Medical Nutritional Therapy 1 Decreases With Age Above target range Within target range Below target range 100% 4% 4% 1% 1% 2% 3% 31% 80% 49% 41% 60% 70% 73% 84% 40% 67% Patients 58% 20% 50% 27% 12% 24% 0% 0-4 Years (N=625) 5-12 Years (N=900) 13-17 Years (N=608) 18-29 Years (N=658) ≥ 30 years (N=687) Pregnant/Planning on Becoming Pregnant (N=106) 0-4 y 5-12 y 13-17 y 18-29 y ≥30 y Pregnant/ (N = 625) (N = 900) (N = 608) (N = 658) (N = 687) Planning Pregnancy Many adult patients with PKU are (N = 106) not followed at metabolic clinics 1. Jurecki ER et al. Mol Genet Metab. 2017;120:190-197. Children With PKU on Diet Have Significantly Lower IQ Than Unaffected Peers and Sibling Controls1,2 120 P < .0001 120 P = .001 112 110 110 107 102 100 100 100 90 90 Peers PKU 80 80 Siblings PKU n = 21 a n = 26b n = 55 c Peers Scale Intelligence Wechsler PKU Wechsler
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