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Multicenter Study of Long-Term Outcomes and Quality of Life in PHACE Syndrome after Age 10.
Braun, Mitchell; Frieden, Ilona J; Siegel, Dawn H; George, Elizabeth; Hess, Christopher P; Fox, Christine K; Chamlin, Sarah L; Drolet, Beth A; Metry, Denise; Pope, Elena; Powell, Julie; Holland, Kristen; Ulschmid, Caden; Liang, Marilyn G; Barry, Kelly K; Ho, Tina; Cotter, Chantal; Baselga, Eulalia; Bosquez, David; Jain, Surabhi Neerendranath; Bui, Jordan K; Lara-Corrales, Irene; Funk, Tracy; Small, Alison; Baghoomian, Wenelia; Yan, Albert C; Treat, James R; Hogrogian, Griffin Stockton; Huang, Charles; Haggstrom, Anita; List, Mary; McCuaig, Catherine C; Barrio, Victoria; Mancini, Anthony J; Lawley, Leslie P; Grunnet-Satcher, Kerrie; Horii, Kimberly A; Newell, Brandon; Nopper, Amy; Garzon, Maria C; Scollan, Margaret E; Mathes, Erin F.
Afiliação
  • Braun M; University of California San Francisco, School of Medicine, San Francisco, CA; Department of Dermatology, University of California San Francisco, San Francisco, CA.
  • Frieden IJ; Department of Dermatology, University of California San Francisco, San Francisco, CA.
  • Siegel DH; Department of Dermatology, Stanford University, Palo Alto, CA.
  • George E; Department of Radiology and Biomedical Imaging, University of California San Francisco, San Francisco, CA.
  • Hess CP; Department of Radiology and Biomedical Imaging, University of California San Francisco, San Francisco, CA.
  • Fox CK; Department of Neurology and Pediatrics, University of California San Francisco, San Francisco, CA.
  • Chamlin SL; Department of Dermatology, Lurie Children's Hospital, Northwestern University Feinberg School of Medicine, Chicago, IL.
  • Drolet BA; Department of Dermatology, University of Wisconsin Madison, Madison, WI.
  • Metry D; Department of Dermatology, Texas Children's Hospital, Baylor College of Medicine, Houston, TX.
  • Pope E; Division of Pediatric Dermatology, Hospital for Sick Children, Temerty Faculty of Medicine, University of Toronto, Toronto, Canada.
  • Powell J; Division of Dermatology, Department of Pediatrics, Sainte-Justine University Hospital Center, University of Montreal, Montreal, Quebec, Canada.
  • Holland K; Department of Dermatology, Medical College of Wisconsin, Milwaukee, WI.
  • Ulschmid C; Department of Dermatology, Medical College of Wisconsin, Milwaukee, WI.
  • Liang MG; Department of Dermatology, Boston Children's Hospital, Harvard Medical School, Boston, MA.
  • Barry KK; Department of Dermatology, Boston Children's Hospital, Harvard Medical School, Boston, MA.
  • Ho T; Department of Dermatology, Boston Children's Hospital, Harvard Medical School, Boston, MA.
  • Cotter C; Department of Dermatology, Boston Children's Hospital, Harvard Medical School, Boston, MA.
  • Baselga E; Department of Dermatology, Hospital de la Sant Pau, Barcelona, Spain.
  • Bosquez D; Department of Dermatology, Hospital de la Sant Pau, Barcelona, Spain.
  • Jain SN; Department of Dermatology, Stanford University, Palo Alto, CA.
  • Bui JK; Department of Dermatology, Stanford University, Palo Alto, CA.
  • Lara-Corrales I; Division of Pediatric Dermatology, Hospital for Sick Children, Temerty Faculty of Medicine, University of Toronto, Toronto, Canada.
  • Funk T; Departments of Dermatology and Pediatrics, Oregon Health & Science University, Portland, OR.
  • Small A; Departments of Dermatology and Pediatrics, Oregon Health & Science University, Portland, OR.
  • Baghoomian W; Departments of Dermatology and Pediatrics, Oregon Health & Science University, Portland, OR.
  • Yan AC; Department of Pediatrics and Dermatology, Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA.
  • Treat JR; Department of Pediatrics and Dermatology, Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA.
  • Hogrogian GS; Department of Pediatrics and Dermatology, Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA.
  • Huang C; Department of Pediatrics and Dermatology, Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA.
  • Haggstrom A; Department of Dermatology, Indiana University School of Medicine, Indianapolis, IN.
  • List M; Department of Dermatology, Indiana University School of Medicine, Indianapolis, IN.
  • McCuaig CC; Division of Dermatology, Department of Pediatrics, Sainte-Justine University Hospital Center, University of Montreal, Montreal, Quebec, Canada.
  • Barrio V; Department of Dermatology, Rady Children's Hospital, University of California San Diego, San Diego, CA.
  • Mancini AJ; Department of Dermatology, Lurie Children's Hospital, Northwestern University Feinberg School of Medicine, Chicago, IL.
  • Lawley LP; Department of Dermatology, Emory University School of Medicine, Atlanta, GA.
  • Grunnet-Satcher K; Department of Dermatology, Emory University School of Medicine, Atlanta, GA.
  • Horii KA; Division of Dermatology, Children's Mercy Hospital and Clinics, Kansas City, MO.
  • Newell B; Division of Dermatology, Children's Mercy Hospital and Clinics, Kansas City, MO.
  • Nopper A; Division of Dermatology, Children's Mercy Hospital and Clinics, Kansas City, MO.
  • Garzon MC; Department of Dermatology, Columbia University Vagelos College of Physicians and Surgeons, New York, NY.
  • Scollan ME; Department of Dermatology, Columbia University Vagelos College of Physicians and Surgeons, New York, NY.
  • Mathes EF; Department of Dermatology, University of California San Francisco, San Francisco, CA. Electronic address: erin.mathes@ucsf.edu.
J Pediatr ; 267: 113907, 2024 Apr.
Article em En | MEDLINE | ID: mdl-38218370
ABSTRACT

OBJECTIVE:

To characterize long-term outcomes of PHACE syndrome. STUDY

DESIGN:

Multicenter study with cross-sectional interviews and chart review of individuals with definite PHACE syndrome ≥10 years of age. Data from charts were collected across multiple PHACE-related topics. Data not available in charts were collected from patients directly. Likert scales were used to assess the impact of specific findings. Patient-Reported Outcomes Measurement Information System (PROMIS) scales were used to assess quality of life domains.

RESULTS:

A total of 104/153 (68%) individuals contacted participated in the study at a median of 14 years of age (range 10-77 years). There were infantile hemangioma (IH) residua in 94.1%. Approximately one-half had received laser treatment for residual IH, and the majority (89.5%) of participants were satisfied or very satisfied with the appearance. Neurocognitive manifestations were common including headaches/migraines (72.1%), participant-reported learning differences (45.1%), and need for individualized education plans (39.4%). Cerebrovascular arteriopathy was present in 91.3%, with progression identified in 20/68 (29.4%) of those with available follow-up imaging reports. Among these, 6/68 (8.8%) developed moyamoya vasculopathy or progressive stenoocclusion, leading to isolated circulation at or above the level of the circle of Willis. Despite the prevalence of cerebrovascular arteriopathy, the proportion of those with ischemic stroke was low (2/104; 1.9%). PROMIS global health scores were lower than population norms by at least 1 SD.

CONCLUSIONS:

PHACE syndrome is associated with long-term, mild to severe morbidities including IH residua, headaches, learning differences, and progressive arteriopathy. Primary and specialty follow-up care is critical for PHACE patients into adulthood.
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Texto completo: 1 Bases de dados: MEDLINE Assunto principal: Coartação Aórtica / Anormalidades do Olho / Síndromes Neurocutâneas Tipo de estudo: Clinical_trials / Prognostic_studies / Risk_factors_studies Limite: Adolescent / Adult / Aged / Child / Humans / Infant / Middle aged Idioma: En Revista: J Pediatr Ano de publicação: 2024 Tipo de documento: Article

Texto completo: 1 Bases de dados: MEDLINE Assunto principal: Coartação Aórtica / Anormalidades do Olho / Síndromes Neurocutâneas Tipo de estudo: Clinical_trials / Prognostic_studies / Risk_factors_studies Limite: Adolescent / Adult / Aged / Child / Humans / Infant / Middle aged Idioma: En Revista: J Pediatr Ano de publicação: 2024 Tipo de documento: Article