Research Output

2020

Safety, Tolerability, and Efficacy of Viltolarsen in Boys with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A Phase 2 Randomized Clinical Trial

Clemens, P. R., Rao, V. K., Connolly, A. M., Harper, A. D., Mah, J. K., Smith, E. C., McDonald, C. M., Zaidman, C. M., Morgenroth, L. P., Osaki, H., Satou, Y., Yamashita, T. & Hoffman, E. P., Jan 1 2020, (Accepted/In press) In : JAMA Neurology.

Research output: Contribution to journalArticle

Open Access
1 Scopus citations

Safety and feasibility of hematopoietic progenitor stem cell collection by mobilization with plerixafor followed by apheresis vs bone marrow harvest in patients with sickle cell disease in the multi-center HGB-206 trial

Tisdale, J. F., Pierciey, F. J., Bonner, M., Thompson, A. A., Krishnamurti, L., Mapara, M. Y., Kwiatkowski, J. L., Shestopalov, I., Ribeil, J. A., Huang, W., Asmal, M., Kanter, J. & Walters, M. C., Jan 1 2020, (Accepted/In press) In : American Journal of Hematology.

Research output: Contribution to journalLetter

Open Access

Safe Work-Hour Standards for Parents of Children with Medical Complexity

Schall, T. E., Foster, C. C. & Feudtner, C., Jan 2020, In : JAMA Pediatrics. 174, 1, p. 7-8 2 p.

Research output: Contribution to journalComment/debate

1 Scopus citations

Screening for prevention of sudden death in the young: What is new?

Webster, G., Carberry, T. & Berger, S., Jan 1 2020, In : Current opinion in cardiology. 35, 1, p. 80-86 7 p.

Research output: Contribution to journalReview article

SENTINEL1: Two-Season Study of Respiratory Syncytial Virus Hospitalizations among US. Infants Born at 29 to 35 Weeks' Gestational Age Not Receiving Immunoprophylaxis

Anderson, E. J., Devincenzo, J. P., Simões, E. A. F., Krilov, L. R., Forbes, M. L., Pannaraj, P. S., Espinosa, C. M., Welliver, R. C., Wolkoff, L. I., Yogev, R., Checchia, P. A., Domachowske, J. B., Halasa, N., McBride, S. J., Kumar, V. R., McLaurin, K. K., Rizzo, C. P. & Ambrose, C. S., Mar 1 2020, In : American journal of perinatology. 37, 4, p. 421-429 9 p.

Research output: Contribution to journalArticle

Open Access
2 Scopus citations

Serum IL-6: A biomarker in childhood asthma?

Jackson, D. J., Bacharier, L. B., Calatroni, A., Gill, M. A., Hu, J., Liu, A. H., Wheatley, L. M., Gern, J. E., Gruchalla, R. S., Khurana Hershey, G. K., Kattan, M., Kercsmar, C. M., Kim, H., O'Connor, G. T., Patel, S., Pongracic, J. A., Wood, R. A. & Busse, W. W., Jan 1 2020, (Accepted/In press) In : Journal of Allergy and Clinical Immunology.

Research output: Contribution to journalArticle

Open Access

Should Decision Making Be Shared in High-Risk Pediatric Heart Donation?

Lelkes, E., Patel, A., Joong, A. G-M. & Gossett, J. G., May 1 2020, In : AMA Journal of Ethics. 22, 5, p. E401-E407

Research output: Contribution to journalArticle

Open Access

Skin-interfaced biosensors for advanced wireless physiological monitoring in neonatal and pediatric intensive-care units

Chung, H. U., Rwei, A. Y., Hourlier-Fargette, A., Xu, S., Lee, K. H., Dunne, E. C., Xie, Z., Liu, C., Carlini, A., Kim, D. H., Ryu, D., Kulikova, E., Cao, J., Odland, I. C., Fields, K. B., Hopkins, B., Banks, A., Ogle, C., Grande, D., Park, J. B. & 42 others, Kim, J., Irie, M., Jang, H., Lee, J. H., Park, Y., Kim, J., Jo, H. H., Hahm, H., Avila, R., Xu, Y., Namkoong, M., Kwak, J. W., Suen, E., Paulus, M. A., Kim, R. J., Parsons, B. V., Human, K. A., Kim, S. S., Patel, M., Reuther, W., Kim, H. S., Lee, S. H., Leedle, J. D., Yun, Y., Rigali, S., Son, T., Jung, I., Arafa, H., Soundararajan, V. R., Ollech, A., Shukla, A., Bradley, A., Schau, M., Rand, C. M., Marsillio, L. E., Harris, Z. L., Huang, Y., Hamvas, A., Paller, A. S., Weese-Mayer, D. E., Lee, J. Y. & Rogers, J. A., Mar 1 2020, In : Nature Medicine. 26, 3, p. 418-429 12 p.

Research output: Contribution to journalArticle

1 Scopus citations

Social Determinants of Cardiovascular Health: Early Life Adversity as a Contributor to Disparities in Cardiovascular Diseases

Suglia, S. F., Campo, R. A., Brown, A. G. M., Stoney, C., Boyce, C. A., Appleton, A. A., Bleil, M. E., Boynton-Jarrett, R., Dube, S. R., Dunn, E. C., Ellis, B. J., Fagundes, C. P., Heard-Garris, N. J., Jaffee, S. R., Johnson, S. B., Mujahid, M. S., Slopen, N., Su, S. & Watamura, S. E., Apr 2020, In : Journal of Pediatrics. 219, p. 267-273 7 p.

Research output: Contribution to journalComment/debate

Open Access

Society of pediatric liver transplantation: Current registry status 2011-2018

the Society of Pediatric Liver Transplantation Research Group, Feb 1 2020, In : Pediatric transplantation. 24, 1, e13605.

Research output: Contribution to journalArticle

1 Scopus citations

Spinal muscular atrophy care in the COVID-19 pandemic era

Veerapandiyan, A., Connolly, A. M., Finkel, R. S., Arya, K., Mathews, K. D., Smith, E. C., Castro, D., Butterfield, R. J., Parsons, J. A., Servais, L., Kuntz, N., Rao, V. K., Brandsema, J. F., Mercuri, E. & Ciafaloni, E., Jul 1 2020, In : Muscle and Nerve. 62, 1, p. 46-49 4 p.

Research output: Contribution to journalArticle

Open Access
1 Scopus citations

Spontaneous Generation and an Eighteenth Century Italian Rabbi-Physician

Katz, B. Z., Jan 1 2020, (Accepted/In press) In : Pediatric Infectious Disease Journal. p. 1228-1229 2 p.

Research output: Contribution to journalReview article

SSBP1 mutations cause mtDNA depletion underlying a complex optic atrophy disorder

Del Dotto, V., Ullah, F., Di Meo, I., Magini, P., Gusic, M., Maresca, A., Caporali, L., Palombo, F., Tagliavini, F., Baugh, E. H., MacAo, B., Szilagyi, Z., Peron, C., Gustafson, M. A., Khan, K., La Morgia, C., Barboni, P., Carbonelli, M., Valentino, M. L., Liguori, R. & 27 others, Shashi, V., Sullivan, J., Nagaraj, S., El-Dairi, M., Iannaccone, A., Cutcutache, I., Bertini, E., Carrozzo, R., Emma, F., Diomedi-Camassei, F., Zanna, C., Armstrong, M., Page, M., Stong, N., Boesch, S., Kopajtich, R., Wortmann, S., Sperl, W., Davis, E. E., Copeland, W. C., Seri, M., Falkenberg, M., Prokisch, H., Katsanis, N., Tiranti, V., Pippucci, T. & Carelli, V., Jan 2 2020, In : Journal of Clinical Investigation. 130, 1, p. 108-125 18 p.

Research output: Contribution to journalArticle

Open Access
4 Scopus citations

Stay the Course: Targeted Evaluation, Accurate Diagnosis, and Treatment of Streptococcal Pharyngitis Prevent Acute Rheumatic Fever

Tanz, R. R., Gewitz, M. H., Kaplan, E. L. & Shulman, S. T., Jan 2020, In : Journal of Pediatrics. 216, p. 208-212 5 p.

Research output: Contribution to journalComment/debate

Sticking by an imperfect standard: Chest radiography for pediatric community-acquired pneumonia

Florin, T. A. & Gerber, J. S., Mar 1 2020, In : Pediatrics. 145, 3, e20193900.

Research output: Contribution to journalComment/debate

Study 275: Updated Expanded Access Program for Remestemcel-L in Steroid-Refractory Acute Graft-versus-Host Disease in Children

for the MSB-275 Study Group, May 2020, In : Biology of Blood and Marrow Transplantation. 26, 5, p. 855-864 10 p.

Research output: Contribution to journalArticle

Open Access
2 Scopus citations

Success of Pediatric Intubations Performed by a Critical Care Transport Service

Ramgopal, S., Button, S. E., Owusu-Ansah, S., Manole, M. D., Saladino, R. A., Guyette, F. X. & Martin-Gill, C., Jan 1 2020, (Accepted/In press) In : Prehospital Emergency Care.

Research output: Contribution to journalArticle

1 Scopus citations

Surrogate Decision Making for Children: Who Should Decide?

Fishman, M., Paquette, E. T., Gandhi, R., Pendergrast, T. R., Park, M., Flanagan, E. & Ross, L. F., May 2020, In : Journal of Pediatrics. 220, p. 221-226 6 p.

Research output: Contribution to journalArticle

2 Scopus citations

Survival of Patients With Non-Rhabdomyosarcoma Soft Tissue Sarcomas of the Head and Neck

Rastatter, J. C., Sinard, R. N., Dilger, A., Reichek, J. L., Walterhouse, D. O. & Patel, U., Jan 1 2020, (Accepted/In press) In : Laryngoscope.

Research output: Contribution to journalArticle

Symptom burden trajectories experienced by patients with brain tumors

Lai, J. S., Beaumont, J. L., Kupst, M. J., Peipert, J. D., Cella, D., Fisher, A. P. & Goldman, S., Jan 1 2020, (Accepted/In press) In : Cancer. 126, 14, p. 3341-3351 11 p.

Research output: Contribution to journalArticle

TCTEX1D1 is a genetic modifier of disease progression in Duchenne muscular dystrophy

CINRG Investigators, Jan 1 2020, (Accepted/In press) In : European Journal of Human Genetics.

Research output: Contribution to journalArticle

Open Access

The care of patients with Duchenne, Becker, and other muscular dystrophies in the COVID-19 pandemic

Veerapandiyan, A., Wagner, K. R., Apkon, S., McDonald, C. M., Mathews, K. D., Parsons, J. A., Wong, B. L., Eichinger, K., Shieh, P. B., Butterfield, R. J., Rao, V. K., Smith, E. C., Proud, C. M., Connolly, A. M. & Ciafaloni, E., Jul 1 2020, In : Muscle and Nerve. 62, 1, p. 41-45 5 p.

Research output: Contribution to journalArticle

Open Access
1 Scopus citations

The Creation of a Pediatric Health Care Learning Network: The ACTION Quality Improvement Collaborative

Lorts, A., Smyth, L., Gajarski, R. J., Vanderpluym, C. J., Mehegan, M., Villa, C. R., Murray, J. M., Niebler, R. A., Almond, C. S., Thrush, P., O'Connor, M. J., Conway, J., Sutcliffe, D. L., Lantz, J. E., Zafar, F., Morales, D. L. S., Peng, D. M. & Rosenthal, D. N., Jan 1 2020, (Accepted/In press) In : ASAIO Journal. p. 441-446 6 p.

Research output: Contribution to journalArticle

1 Scopus citations

The Future of Vascular Access in Children

Schroeder, S. K. & Davis, M. M., Jun 1 2020, In : Pediatrics. 145, p. S300-S301

Research output: Contribution to journalArticle

Open Access

The gut of healthy infants in the community as a reservoir of esbl and carbapenemase- producing bacteria

Saleem, A. F., Allana, A., Hale, L., Diaz, A., Salinas, R., Salinas, C., Qureshi, S. M., Hotwani, A., Rahman, N., Khan, A., Zaidi, A. K., Seed, P. C. & Arshad, M., Jun 2020, In : Antibiotics. 9, 6, 286.

Research output: Contribution to journalArticle

Open Access

The Human Tumor Atlas Network: Charting Tumor Transitions across Space and Time at Single-Cell Resolution

Human Tumor Atlas Network, Apr 16 2020, In : Cell. 181, 2, p. 236-249 14 p.

Research output: Contribution to journalReview article

Open Access
1 Scopus citations

The impact of gun violence on those already dying: Perspectives from a palliative care physician

Vente, T. M., Feb 1 2020, In : Pediatrics. 145, 2

Research output: Contribution to journalReview article

The impact of placental pathology discordance in multiple gestation pregnancies on bronchopulmonary dysplasia-associated pulmonary hypertension

Franklin, A., Yallapragada, S. G., Birkett, R., Grobman, W., Ernst, L. M. & Mestan, K., Jan 1 2020, In : Pulmonary Circulation. 10, 1

Research output: Contribution to journalArticle

Open Access

The Joint Associations of Maternal BMI and Glycemia with Childhood Adiposity

Josefson, J. L., Catalano, P. M., Lowe, W. L., Scholtens, D. M., Kuang, A., Dyer, A. R., Lowe, L. P. & Metzger, B. E., Jul 1 2020, In : The Journal of clinical endocrinology and metabolism. 105, 7

Research output: Contribution to journalArticle

The Prevalence of Pediatric Myalgic Encephalomyelitis/Chronic Fatigue Syndrome in a Community-Based Sample

Jason, L. A., Katz, B. Z., Sunnquist, M., Torres, C., Cotler, J. & Bhatia, S., Jan 1 2020, (Accepted/In press) In : Child and Youth Care Forum.

Research output: Contribution to journalArticle

1 Scopus citations

The promise and pitfalls of precision medicine to resolve black–white racial disparities in preterm birth

Burris, H. H., Wright, C. J., Kirpalani, H., Collins, J. W., Lorch, S. A., Elovitz, M. A. & Hwang, S. S., Jan 1 2020, In : Pediatric research. 87, 2, p. 221-226 6 p.

Research output: Contribution to journalReview article

The pyruvate kinase (PK) to hexokinase enzyme activity ratio and erythrocyte PK protein level in the diagnosis and phenotype of PK deficiency

Al-Samkari, H., Addonizio, K., Glader, B., Morton, D. H., Chonat, S., Thompson, A. A., Kuo, K. H. M., Ravindranath, Y., Wang, H., Rothman, J. A., Kwiatkowski, J. L., Kung, C., Kosinski, P. A., Al-Sayegh, H., London, W. B. & Grace, R. F., Jan 1 2020, (Accepted/In press) In : British Journal of Haematology.

Research output: Contribution to journalArticle

Therapies that enhance pulmonary vascular NO-signaling in the neonate

Dillard, J., Perez, M. & Chen, B., Feb 1 2020, In : Nitric Oxide - Biology and Chemistry. 95, p. 45-54 10 p.

Research output: Contribution to journalReview article

The Skin–Immune System Interface

Jhaveri, R., Jan 1 2020, (Accepted/In press) In : Clinical Therapeutics.

Research output: Contribution to journalEditorial

Open Access

Topical calcineurin inhibitors for pediatric periorificial dermatitis

Ollech, A., Yousif, R., Kruse, L., Wagner, A., Kenner-Bell, B., Chamlin, S., Yun, D., Shen, L., Vivar, K., Reynolds, M., Paller, A. S. & Mancini, A. J., Jun 2020, In : Journal of the American Academy of Dermatology. 82, 6, p. 1409-1414 6 p.

Research output: Contribution to journalArticle

Topical cholesterol/lovastatin for the treatment of porokeratosis: A pathogenesis-directed therapy

Atzmony, L., Lim, Y. H., Hamilton, C., Leventhal, J. S., Wagner, A., Paller, A. S. & Choate, K. A., Jan 2020, In : Journal of the American Academy of Dermatology. 82, 1, p. 123-131 9 p.

Research output: Contribution to journalArticle

4 Scopus citations

Trajectory of mortality and health-related quality of life morbidity following community-acquired pediatric septic shock

the Life After Pediatric Sepsis Evaluation (LAPSE) Investigators, Jan 1 2020, (Accepted/In press) In : Critical care medicine. p. 329-337 9 p.

Research output: Contribution to journalArticle

2 Scopus citations

Transforaminal Intrathecal Access for Injection of Nusinersen in Adult and Pediatric Patients with Spinal Muscular Atrophy

Shokuhfar, T., Abdalla, R. N., Hurley, M. C., Nazari, P., Ansari, S. A., Ajroud-Driss, S., Kuntz, N., Azmi, S., Rao, V. & Shaibani, A., Apr 1 2020, In : Journal of Pediatric Neurology. 18, 2, p. 88-94 7 p.

Research output: Contribution to journalArticle

1 Scopus citations

Trends in healthcare utilization for infantile haemangioma in the U.S.A.

Singh, P., Chamlin, S., Hignett, E. & Silverberg, J. I., Feb 1 2020, In : British Journal of Dermatology. 182, 2, p. 509-511 3 p.

Research output: Contribution to journalLetter

Turner Syndrome Systematic Review: Spontaneous Thelarche and Menarche Stratified by Karyotype

Dabrowski, E., Jensen, R., Johnson, E. K., Habiby, R. L., Brickman, W. J. & Finlayson, C., Feb 1 2020, In : Hormone Research in Paediatrics. 92, 3, p. 143-149 7 p.

Research output: Contribution to journalReview article

Turner Syndrome with Y Chromosome: Spontaneous Thelarche, Menarche, and Risk of Malignancy

Dabrowski, E., Johnson, E. K., Patel, V., Hsu, Y. C., Davis, S., Goetsch, A. L., Habiby, R., Brickman, W. J. & Finlayson, C., Feb 2020, In : Journal of pediatric and adolescent gynecology. 33, 1, p. 10-14 5 p.

Research output: Contribution to journalArticle

1 Scopus citations

Tutorial: Nutrition Therapy in Eosinophilic Esophagitis—Outcomes and Deficiencies

Bashaw, H., Schwartz, S., Kagalwalla, A. F. & Wechsler, J. B., May 1 2020, In : Journal of Parenteral and Enteral Nutrition. 44, 4, p. 600-609 10 p.

Research output: Contribution to journalReview article

Understanding Brain Death

Truog, R. D., Paquette, E. T. & Tasker, R. C., Jun 2 2020, In : JAMA - Journal of the American Medical Association. 323, 21, p. 2139-2140 2 p.

Research output: Contribution to journalArticle