Lupus Science and Medicine (Feb 2021)
Delivering clinical trials at home: protocol, design and implementation of a direct-to-family paediatric lupus trial
- E Schmitt,
- C Smith,
- G Schulert,
- S Canna,
- A Grom,
- E Mellins,
- A Brown,
- A Stevens,
- M Watson,
- K Stewart,
- E Baker,
- A Kemp,
- T Davis,
- A Smith,
- S Jackson,
- K Jones,
- T Mason,
- A Hanson,
- Y Zhao,
- J Jones,
- J Chang,
- M Holland,
- K Moore,
- B ferreira,
- H Schmeling,
- K Abulaban,
- R Agbayani,
- S Akoghlanian,
- E Anderson,
- L Barillas-Arias,
- K Baszis,
- M Becker,
- H Bell-Brunson,
- H Benham,
- S Benseler,
- T Beukelman,
- H Brunner,
- H Bukulmez,
- L Cerracchio,
- E Chalom,
- K Chundru,
- J Dean,
- F Dedeoglu,
- V Dempsey,
- J Drew,
- B Feldman,
- P Ferguson,
- C Fleming,
- L Franco,
- I Goh,
- D Goldsmith,
- B Gottlieb,
- T Graham,
- T Griffin,
- M Hance,
- K Hickey,
- M Hollander,
- J Hsu,
- A Huber,
- C Hung,
- A Huttenlocher,
- L Imundo,
- C Inman,
- J Jaquith,
- L Jung,
- D Kingsbury,
- K Klein,
- M Klein-Gitelman,
- S Kramer,
- S Lapidus,
- D Latham,
- B Malla,
- M Malloy,
- A Martyniuk,
- K McConnell,
- D McCurdy,
- C McMullen-Jackson,
- L Moorthy,
- E Muscal,
- J Olson,
- K Onel,
- L Ponder,
- S Prahalad,
- C Rabinovich,
- S Ringold,
- M Riordan,
- A Robinson,
- M Rosenkranz,
- B Rosolowski,
- N Ruth,
- K Schikler,
- H Stapp,
- R Syed,
- M Tesher,
- A Thatayatikom,
- R Vehe,
- E von Scheven,
- D Wahezi,
- A Watts,
- J Weiss,
- S Kim,
- J Patel,
- J Stokes,
- L Marques,
- Stephen J Balevic,
- D Lovell,
- A Zeft,
- J Harris,
- E Lawson,
- C Moss,
- N George,
- Rachel L Randell,
- M Adams,
- S Cooper,
- M Miller,
- C Black,
- R Schneider,
- J Taylor,
- R Sran,
- M Oliver,
- M Twilt,
- M Tóth,
- J Walker,
- M Mitchell,
- F De Benedetti,
- N Singer,
- M Fox,
- K Kaufman,
- A Merritt,
- R Stevenson,
- J Fuller,
- M Fitzgerald,
- A Davis,
- C Davis,
- L Henderson,
- J Woo,
- S Mohan,
- H Reid,
- Y Kimura,
- L Harel,
- R Laxer,
- K McCarthy,
- I Ferguson,
- E McCormick,
- M Guzman,
- P Hill,
- A PARSONS,
- S McGuire,
- J Lam,
- C Sandborg,
- B Stevens,
- J Boland,
- S Ballinger,
- E MENDOZA,
- J NOCTON,
- M Ritter,
- N Johnson,
- J Shirley,
- S Bowman,
- M Ibarra,
- S Hong,
- M Guevara,
- K James,
- L Santiago,
- A Adams,
- B DONALDSON,
- M Son,
- C Kremer,
- K Schmidt,
- T Wright,
- L Cannon,
- R Nicolai,
- M Freeman,
- S Spence,
- D Levy,
- J Paredes,
- K Gerhold,
- A Insalaco,
- T O'Brien,
- W Bernal,
- E Kessler,
- C Lin,
- M Lerman,
- T Hahn,
- B O'Brien,
- Lindsay Singler,
- Anthony Cunningham,
- Michael Cohen-Wolkowiez,
- Christoph P Hornik,
- N Abel,
- J Aiello,
- C Alejandro,
- E Allenspach,
- R Alperin,
- M Alpizar,
- G Amarilyo,
- W Ambler,
- S Ardoin,
- S Armendariz,
- I Balboni,
- S Balevic,
- L Ballenger,
- N Balmuri,
- F Barbar-Smiley,
- M Basiaga,
- E Beltz,
- T Bigley,
- B Binstadt,
- M Blakley,
- J Bohnsack,
- A Boneparth,
- C Bracaglia,
- E Brooks,
- M Brothers,
- M Buckley,
- D Bullock,
- B Cameron,
- P Carper,
- V Cartwright,
- E Cassidy,
- A Chang-Hoftman,
- V Chauhan,
- P Chira,
- T Chinn,
- H Clairman,
- D Co,
- A Confair,
- H Conlon,
- R Connor,
- C Correll,
- R Corvalan,
- D Costanzo,
- R Cron,
- L Curiel-Duran,
- T Curington,
- M Curry,
- A Dalrymple,
- D De Ranieri,
- M De Guzman,
- N Delnay,
- E DeSantis,
- T Dickson,
- J Dingle,
- E Dorsey,
- S Dover,
- J Dowling,
- K Driest,
- Q Du,
- K Duarte,
- D Durkee,
- E Duverger,
- J Dvergsten,
- A Eberhard,
- M Eckert,
- K Ede,
- B Edelheit,
- C Edens,
- Y Edgerly,
- M Elder,
- B Ervin,
- S Fadrhonc,
- C Failing,
- D Fair,
- M Falcon,
- S Federici,
- J Fennell,
- R Ferrucho,
- K Fields,
- T Finkel,
- O Flynn,
- L Fogel,
- K Fritz,
- S Froese,
- R Fuhlbrigge,
- D Gerstbacher,
- M Gilbert,
- M Gillispie-Taylor,
- E Giverc,
- C Godiwala,
- H Goheer,
- E Gotschlich,
- A Gotte,
- C Gracia,
- S Grevich,
- J Griswold,
- P Guittar,
- M Hager,
- O Halyabar,
- E Hammelev,
- S Haro,
- O Harry,
- E Hartigan,
- J Hausmann,
- J Heiart,
- K Hekl,
- M Henrickson,
- A Hersh,
- S Hillyer,
- L Hiraki,
- M Hiskey,
- P Hobday,
- C Hoffart,
- M Horwitz,
- J Huggins,
- J HuiYuen,
- J Huntington,
- G Janow,
- S Jared,
- C Justice,
- A Justiniano,
- N Karan,
- U Khalsa,
- B Kienzle,
- M Kitcharoensakkul,
- T Klausmeier,
- B Kompelien,
- A Kosikowski,
- L Kovalick,
- J Kracker,
- J Lai,
- B Lang,
- B Lapin,
- A Lasky,
- L Lentini,
- S Lieberman,
- N Ling,
- M Lingis,
- M Lo,
- D Lowman,
- N Luca,
- S Lvovich,
- C Madison,
- J Madison,
- S Magni Manzoni,
- J Maller,
- M Mannion,
- C Manos,
- S Mathus,
- L McAllister,
- P McCurdy Stokes,
- I McHale,
- A McMonagle,
- E Meidan,
- R Mercado,
- L Michalowski,
- P Miettunen,
- D Milojevic,
- E Mirizio,
- E Misajon,
- R Modica,
- E Morgan Dewitt,
- T Moussa,
- V Mruk,
- R Nadler,
- B Nahal,
- K Nanda,
- N Nasah,
- L Nassi,
- S Nativ,
- M Natter,
- J Neely,
- B Nelson,
- L Newhall,
- L Ng,
- P Nigrovic,
- B Nolan,
- E Oberle,
- B Obispo,
- O Okeke,
- K O'Neil,
- A Orandi,
- M Orlando,
- S Osei-Onomah,
- R Oz,
- A Paller,
- N Pan,
- S Panupattanapong,
- M Pardeo,
- K Pentakota,
- P Pepmueller,
- T Pfeiffer,
- K Phillippi,
- D Pires Marafon,
- R Pooni,
- S Pratt,
- S Protopapas,
- B Puplava,
- J Quach,
- M Quinlan-Waters,
- S Radhakrishna,
- J Rafko,
- J Raisian,
- A Rakestraw,
- E Ramsay,
- S Ramsey,
- R Randell,
- K Remmel,
- A Repp,
- A Reyes,
- A Richmond,
- M Riebschleger,
- M Riskalla,
- R Rivas-Chacon,
- E Rodela,
- M Rodriquez,
- K Rojas,
- T Ronis,
- H Rothermel,
- D Rothman,
- E Roth-Wojcicki,
- K Rouster-Stevens,
- T Rubinstein,
- N Saad,
- S Sabbagh,
- E Sacco,
- R Sadun,
- A Sanni,
- A Sarkissian,
- S Savani,
- L Scalzi,
- L Schanberg,
- S Scharnhorst,
- A Schlefman,
- K Schollaert-Fitch,
- T Seay,
- C Seper,
- J Shalen,
- R Sheets,
- A Shelly,
- S Shenoi,
- K Shergill,
- M Shishov,
- C Shivers,
- E Silverman,
- V Sivaraman,
- J Sletten,
- E Smitherman,
- J Soep,
- L Spiegel,
- J Spitznagle,
- H Srinivasalu,
- K Steigerwald,
- Y Sterba Rakovchik,
- S Stern,
- C Stingl,
- M Stoll,
- E Stringer,
- S Sule,
- J Sumner,
- R Sundel,
- G Syverson,
- A Szymanski,
- S Taber,
- R Tal,
- A Tambralli,
- A Taneja,
- T Tanner,
- S Tapani,
- G Tarshish,
- S Tarvin,
- L Tate,
- A Taxter,
- M Terry,
- K Tiffany,
- T Ting,
- A Tipp,
- D Toib,
- K Torok,
- C Toruner,
- H Tory,
- S Tse,
- V Tubwell,
- S Uriguen,
- T Valcarcel,
- H Van Mater,
- L Vannoy,
- C Varghese,
- N Vasquez,
- K Vazzana,
- K Veiga,
- J Velez,
- J Verbsky,
- G Vilar,
- N Volpe,
- S Vora,
- L Wagner-Weiner,
- H Waite,
- H Walters,
- T Wampler Muskardin,
- L Waqar,
- M Waterfield,
- P Weiser,
- P Weiss,
- E Wershba,
- A Wise,
- L Woolnough,
- E Wu,
- A Yalcindag,
- M Yee,
- E Yen,
- R Yeung,
- K Yomogida,
- Q Yu,
- R Zapata,
- A Zartoshti,
- R Zeft,
- A Zhu,
- C Zic
Affiliations
- E Schmitt
- Orthopaedic Clinic Friedrichsheim, Johann Wolfgang Goethe University, Frankfurt/Main, Germany
- C Smith
- Nutrition and Dietetics, Brighton and Sussex University Hospitals NHS Trust, Brighton, UK
- G Schulert
- 4Cincinnati Children’s Hospital, Cincinnati
- S Canna
- 5Children’s Hospital of Pittsburgh, Pittsburgh, USA
- A Grom
- 4Cincinnati Children’s Hospital, Cincinnati
- E Mellins
- 1Stanford University, Palo Alto
- A Brown
- Paediatric Neurology, Nottingham Children's Hospital, Nottingham, UK
- A Stevens
- 1Centre for Paediatrics and Child Health, Institute of Human Development, University of Manchester, Manchester, UK
- M Watson
- 1Paediatrics, Imperial College NHS Trust, London, UK
- K Stewart
- 3Department of Medicine
- E Baker
- PIND Research Group, Addenbrooke’s Hospital, Cambridge, UK
- A Kemp
- 2School of Medicine, Cardiff University, Cardiff, UK
- T Davis
- Paediatric Emergency Department, Royal London Hospital, London, UK
- A Smith
- Imperial College Healthcare NHS Trust, London, UK
- S Jackson
- 1Centre for Academic Child Health, University of Bristol, Bristol, UK
- K Jones
- Bristol Children’s Emergency Department, Bristol Children’s Hospital, Bristol, UK
- T Mason
- Paediatrics, Royal Devon and Exeter, Exeter, UK
- A Hanson
- 4Department of Molecular and Clinical Pharmacology, Institute of Translational Medicine, University of Liverpool
- Y Zhao
- Taipei City Hospital, Pharmacy, Taipei, Taiwan R.O.C
- J Jones
- 1Neonatal Unit, Norfolk and Norwich University Hospitals NHS Foundation Trust, Norwich, UK
- J Chang
- 8Neonatal Services, Croydon University Hospital, London, UK
- M Holland
- Department of Rheumatology and Clinical Immunology, Charité Universitätsmedizin Berlin, Berlin, Germany
- K Moore
- Department of Diabetes, Beaumont Hospital, Dublin, Ireland [email protected]
- B ferreira
- 2FCECON, Clinical Oncology, Manaus, Brazil
- H Schmeling
- Department of Paediatrics, University Medical Centre, Martin Luther University, Halle-Wittenberg, Germany
- K Abulaban
- R Agbayani
- S Akoghlanian
- E Anderson
- Centre for Academic Child Health, University of Bristol, Bristol, UK
- L Barillas-Arias
- K Baszis
- M Becker
- Department of Paediatrics, Charité, Campus Virchow-Klinikum
- H Bell-Brunson
- H Benham
- S Benseler
- T Beukelman
- Division of Clinical Immunology and Rheumatology, University of Alabama at Birmingham, Birmingham, Alabama, USA
- H Brunner
- Department of Internal Medicine, Krankenhaus Lainz, Vienna, Austria
- H Bukulmez
- L Cerracchio
- E Chalom
- K Chundru
- J Dean
- 4University of East Anglia Medical School, Norwich, UK
- F Dedeoglu
- V Dempsey
- J Drew
- Children & Young People’s Kidney Unit, Hucknall Road, Nottingham NG5 1PB, UK
- B Feldman
- P Ferguson
- Heartlands Hospital, Birmingham, UK
- C Fleming
- 4th year medical student
- L Franco
- I Goh
- D Goldsmith
- Intensive Care Unit, Austin Hospital, Melbourne, Victoria, Australia
- B Gottlieb
- T Graham
- 2Centre for Evolution and Cancer, University of California at San Francisco, San Francisco, United States
- T Griffin
- M Hance
- K Hickey
- M Hollander
- Department of Epidemiology and Biostatistics, Erasmus Medical Centre, Rotterdam, Netherlands
- J Hsu
- A Huber
- Department of Medicine and Radiology, University of Munich, Munich, Germany
- C Hung
- A Huttenlocher
- L Imundo
- C Inman
- 2Birmingham City University, UK
- J Jaquith
- L Jung
- D Kingsbury
- K Klein
- 1Center of Experimental Rheumatology, University Hospital Zurich
- M Klein-Gitelman
- S Kramer
- Department of Radiology, University of Münster, Münster, Germany
- S Lapidus
- D Latham
- B Malla
- M Malloy
- 11National Hpv Vaccination Program Register, VCS, Parkville, Australia
- A Martyniuk
- McMaster University, Hamilton, ON, Canada
- K McConnell
- D McCurdy
- C McMullen-Jackson
- L Moorthy
- E Muscal
- J Olson
- K Onel
- L Ponder
- S Prahalad
- Department of Pediatrics, Primary Children’s Hospital and University of Utah, Utah, USA
- C Rabinovich
- S Ringold
- M Riordan
- Department of Pediatrics, Yale University Medical School, USA
- A Robinson
- 1School of Clinical Medicine, University of Cambridge, Cambridge, UK
- M Rosenkranz
- Klinik und Poliklinik für Neurologie, Neuro-Zentrum, Hamburg, Germany
- B Rosolowski
- N Ruth
- Community Child Health, Shropshire Community Trust. Shrewsbury, UK
- K Schikler
- H Stapp
- R Syed
- [email protected]
- M Tesher
- A Thatayatikom
- R Vehe
- E von Scheven
- D Wahezi
- A Watts
- Department of Ophthalmic Surgery, University Hospitals of Leicester NHS Trust, Leicester LE1 5WW, UK
- J Weiss
- Kolling Institute of Medical Research, University of Sydney, Royal North Shore Hospital, St Leonards, New South Wales 2065, Australia
- S Kim
- Rheumatology, The Hospital for Rheumatic Diseases, Seoul, Korea
- J Patel
- 1Emergency Medicine, University Hospitals Leicester, Leicester, UK
- J Stokes
- 1HammondCare, Greenwich, New South Wales, Australia
- L Marques
- 2Beatriz Ângelo Hospital, Medicine Department, Loures, Portugal
- Stephen J Balevic
- 1 Division of Rheumatology, Department of Medicine, Duke Health, Durham, North Carolina, USA
- D Lovell
- 2PRCSG, CHMC, Cincinnati
- A Zeft
- 15Pediatrics Rheumatology, Cleveland Clinic, Cleveland, United States
- J Harris
- Paediatrics, Leicester Royal Infirmary, Leicester, UK
- E Lawson
- 5Paediatric Neurosurgery, Royal Manchester Children’s Hospital, Manchester, UK
- C Moss
- 2Dermatology, Birmingham Women’s and Children’s NHS FT, Birmingham, UK
- N George
- AandE, Homerton University Hospital, London, UK
- Rachel L Randell
- Division of Pediatric Rheumatology, Department of Pediatrics, Duke University, Durham, North Carolina, USA
- M Adams
- 1Department of Women and Children’s Health, King’s College, London, UK
- S Cooper
- 1Department of Applied Health Research, UCL, UK
- M Miller
- Oxford University Hospitals NHS Trust
- C Black
- Royal Free Hospital, London, UK
- R Schneider
- Department of Radiology, Hospital for Special Surgery, New York, USA
- J Taylor
- Department of Paediatric Nephrology, Evelina Children's Hospital, Guy's and St Thomas' Hospitals NHS Trust, London, UK
- R Sran
- 1School of Population and Public Health, Faculty of Medicine, University of British Columbia, Vancouver, Canada
- M Oliver
- 2Department of Epidemiology and Biostatistics, Faculty of Health and Environmental Science, Auckland University of Technology, Auckland, New Zealand
- M Twilt
- 1Rheumatology Department, Birmingham Children’s Hospital, Birmingham, UK
- M Tóth
- 1Department of Immunology, Eötvös Loránd University, Budapest, HU
- J Walker
- Department of Ophthalmology, Great Ormond St Hospital for Children, London, UK
- M Mitchell
- The Hospital for Sick Children, Toronto, Canada
- F De Benedetti
- 14 Laboratorio di Reumatologia, IRCCS Ospedale Pediatrico Bambino Gesù, Rome, Italy
- N Singer
- 19 Division of Pediatric Infectious Disease and Rheumatology Division of Rheumatic Diseases, Department of Medicine Rainbow, Babies and Children's Hospital, Cleveland, Ohio, USA
- M Fox
- 4 Center for International Health and Development, Boston University School of Public Health, Boston, MA, USA
- K Kaufman
- Psychiatry and Neurology, Rutgers Robert Wood Johnson Medical School, New Brunswick, USA
- A Merritt
- 4Department of Pediatrics, Loma Linda University, Loma Linda, USA
- R Stevenson
- 4Department of Pediatrics, University of Virginia, Charlottesville, USA
- J Fuller
- Meyers Primary Care Institute, University of Massachusetts Medical School and Fallon Foundation, Worcester, Massachusetts, USA
- M Fitzgerald
- Radiology Department, Royal Children’s Hospital, Parkville, Melbourne, Australia
- A Davis
- 1University of Washington, Seattle, USA
- C Davis
- 2Newcastle upon Tyne NHS Trust, Freeman Hospital, Freeman Road, NE7 7DN
- L Henderson
- Department of Neurophysiology, Birmingham Children’s Hospital, Birmingham, UK
- J Woo
- Department of Medicine and Therapeutics, The Chinese University of Hong Kong, Shatin, Hong Kong, Hong Kong
- S Mohan
- Musculoskeletal Disease Center, Jerry L Pettis VA Medical Center, Loma Linda, CA, USA
- H Reid
- 1School of Medicine, University College London, London, UK
- Y Kimura
- Department of Rehabilitation, Osaka University Hospital, Osaka, Japan
- L Harel
- Department of Pediatrics C, Schneider Children’s Medical Center of Israel, Petah Tiqva, Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel
- R Laxer
- Department of Pediatrics, The Hospital for Sick Children, Toronto, Canada
- K McCarthy
- Rotunda Hospital, Dublin, Ireland
- I Ferguson
- Department of Gastroenterology, Selly Oak Hospital, Raddlebarn Road, Birmingham B29 6JD, UK
- E McCormick
- 5SHSCT
- M Guzman
- 2Pharmacy Unit, Valme University Hospital, Seville, Spain
- P Hill
- Great Ormond Street Hospital for Children NHS Trust, Department of Psychological Medicine, Great Ormond Street Hospital for Children, Great Ormond Street, London WC1N 3JH, UK; [email protected]
- A PARSONS
- Department of Histopathology, Royal Hallamshire Hospital, Glossop Road, Sheffield S10 2JF, UK
- S McGuire
- J Lam
- Washington University School of Medicine, St Louis, USA
- C Sandborg
- Divisions of Pediatric Cardiology and Rheumatology, Stanford University, California, USA
- B Stevens
- Child Health Evaluative Sciences, Research Institute, The Hospital for Sick Children, Toronto, ON, Canada
- J Boland
- 1Hull York Medical School, University of Hull
- S Ballinger
- E MENDOZA
- 3Facultad de Ciencias Naturales, Exactas y Tecnología, Universidad de Panamá, Panamá, Panamá
- J NOCTON
- M Ritter
- Department of Ophthalmology, Medical University of Vienna, Vienna, Austria
- N Johnson
- 4Children’s Unit, Hinchingbrooke Hospital, Huntingdon, UK
- J Shirley
- 2Clinical Research, Florida Hospital Tampa Bay, Tampa, USA
- S Bowman
- Department of Rheumatology, Birmingham Heartlands and Solihull Hospital NHS Trust, Birmingham, UK
- M Ibarra
- Hospital Universitario Puerta de Hierro, Pharmacy, Madrid, Spain
- S Hong
- The Key Laboratory of AIDS Immunology of Ministry of Health, No 1 Hospital of China Medical University, Shenyang, China
- M Guevara
- Public Health Institute of Navarra, Pamplona-Iruña, Spain
- K James
- University of Colorado, Denver, Colorado, USA
- L Santiago
- Oswaldo Cruz Foundation, National School of Public Health, Rio de Janeiro, Brazil
- A Adams
- 2Warwick Medical School, University of Warwick, Coventry, UK
- B DONALDSON
- M Son
- Department of Preventive Medicine, School of Medicine, Kangwon National University, Korea
- C Kremer
- Department of Neurology, University Hospital of Zürich, Switzerland
- K Schmidt
- Department of Medical Genetics, Molecular and Clinical Pharmacology, Medical University Innsbruck, Austria
- T Wright
- 1NIHR Biomedical Research Unit and Centre for Liver Research, UK
- L Cannon
- Orthopaedic Specialist Registrar
- R Nicolai
- Direzione Scientifica, Sigma-Tau SpA, Pomezia, Italy
- M Freeman
- S Spence
- 1Institute of Health and Society, Newcastle University, Newcastle upon Tyne, UK
- D Levy
- 3University of Georgetown, USA
- J Paredes
- Autoimmune Research Laboratory, Internal Medicine Service, Hospital General Vall d’Hebron, Barcelona, Spain
- K Gerhold
- 3Pediatric Pneumology and Immunology, Charité—Universitätsmedizin Berlin, Berlin, Germany
- A Insalaco
- 12Reumatologia, Ospedale Pediatrico Bambin Gesù, Roma, Italy
- T O'Brien
- Regenerative Medicine Institute, National University of Ireland Galway, Ireland
- W Bernal
- 3Children’s Hospital of Philadelphia Research Institute, Philadelphia, PA, USA
- E Kessler
- C Lin
- Bristol-Myers Squibb, Pennington, New Jersey, USA
- M Lerman
- T Hahn
- B O'Brien
- General practitioner London N18 2ER
- Lindsay Singler
- Pediatrics, Duke Clinical Research Institute, Durham, North Carolina, USA
- Anthony Cunningham
- Pediatrics, Duke Clinical Research Institute, Durham, North Carolina, USA
- Michael Cohen-Wolkowiez
- Pediatrics, Duke University School of Medicine, Durham, North Carolina, USA
- Christoph P Hornik
- Pediatrics, Duke University School of Medicine, Durham, North Carolina, USA
- N Abel
- J Aiello
- C Alejandro
- E Allenspach
- R Alperin
- M Alpizar
- G Amarilyo
- W Ambler
- S Ardoin
- S Armendariz
- I Balboni
- S Balevic
- L Ballenger
- N Balmuri
- F Barbar-Smiley
- M Basiaga
- E Beltz
- T Bigley
- B Binstadt
- M Blakley
- J Bohnsack
- A Boneparth
- C Bracaglia
- E Brooks
- M Brothers
- Neurosurgery, Medical University of South Carolina, South Carolina, USA
- M Buckley
- Department of Gastroenterology, Mercy University Hospital, Cork, Ireland
- D Bullock
- B Cameron
- P Carper
- V Cartwright
- E Cassidy
- A Chang-Hoftman
- V Chauhan
- 2General Surgery, Warwick Hospital, Warwick, UK
- P Chira
- T Chinn
- H Clairman
- D Co
- A Confair
- H Conlon
- R Connor
- C Correll
- R Corvalan
- D Costanzo
- R Cron
- L Curiel-Duran
- T Curington
- M Curry
- A Dalrymple
- D De Ranieri
- M De Guzman
- N Delnay
- E DeSantis
- T Dickson
- J Dingle
- E Dorsey
- Boston Graduate School of Psychoanalysis, Brookline, Massachusetts, USA
- S Dover
- J Dowling
- Northwest Immediate Care Programme, Manorcunningham, County Donegal, Ireland
- K Driest
- Q Du
- K Duarte
- D Durkee
- E Duverger
- J Dvergsten
- A Eberhard
- M Eckert
- Biomolecular Sciences Applied Research Group, Faculty of Health & Life Sciences, Coventry University, Coventry, UK
- K Ede
- B Edelheit
- C Edens
- Y Edgerly
- M Elder
- B Ervin
- S Fadrhonc
- C Failing
- D Fair
- M Falcon
- S Federici
- J Fennell
- Gastrointestinal Unit, Molecular Medicine Centre, University of Edinburgh, Western General Hospital, Edinburgh, UK
- R Ferrucho
- K Fields
- T Finkel
- O Flynn
- L Fogel
- K Fritz
- 2International Center for Research on Women, Washington, DC, USA
- S Froese
- Withybush General Hospital, Haverfordwest, Pembrokeshire SA61 2PZ
- R Fuhlbrigge
- D Gerstbacher
- M Gilbert
- 2BC Centre for Disease Control, Vancouver, Canada
- M Gillispie-Taylor
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- A Gotte
- C Gracia
- S Grevich
- J Griswold
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- M Hager
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- B Kienzle
- M Kitcharoensakkul
- T Klausmeier
- B Kompelien
- A Kosikowski
- L Kovalick
- J Kracker
- J Lai
- School of Health and Related Research, University of Sheffield, Sheffield, UK
- B Lang
- Neurosciences Group, Department of Clinical Neurology, John Radcliffe Hospital, Oxford, UK
- B Lapin
- A Lasky
- L Lentini
- S Lieberman
- N Ling
- M Lingis
- M Lo
- D Lowman
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- L Nassi
- S Nativ
- M Natter
- J Neely
- B Nelson
- L Newhall
- L Ng
- Neurorehabilitation Unit, Royal Melbourne Hospital, Melbourne, Australia
- P Nigrovic
- B Nolan
- Department of Paediatric Haematology, Sheffield Children’s NHS Trust, Sheffield, UK
- E Oberle
- B Obispo
- O Okeke
- K O'Neil
- A Orandi
- M Orlando
- Center for Global Development, Washington, DC, USA
- S Osei-Onomah
- R Oz
- A Paller
- N Pan
- S Panupattanapong
- M Pardeo
- Department of Pediatric Cardiology, Catholic University Medical School, Rome, Italy
- K Pentakota
- P Pepmueller
- T Pfeiffer
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- D Pires Marafon
- R Pooni
- S Pratt
- York Teaching Hospital NHS Foundation Trust, York, UK
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- Murdoch Children's Research Institute, Melbourne, Australia
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- 1School of Medical Sciences, Edith Cowan University, Perth, Australia
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- The Hatter Cardiovascular Institute, University College London, UK
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- Leibniz-Institute for Atherosclerosis Research, University of Muenster, Germany
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- 1Health and Safety Laboratory, Buxton, UK
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- L Wagner-Weiner
- H Waite
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- Department of Medicine and Therapeutics, University of Aberdeen, Aberdeen AB9 2ZD Thoracic Medicine, Aberdeen Royal Hospitals Trust, Aberdeen AB9 8AQ Department of Respiratory Medicine, Alfred Hospital and Monash University, Australia Avondale Medical Practice, Strathaven Health Centre, Strathaven ML10 6AS Newmilns KE16 9EA Greater Glasgow Health Board, Glasgow G2 4JT Information and Statistic Division, NHS in Scotland, Edinburgh EH5 3SQ Luton, Bedfordshire LU4 8SE.
- T Wampler Muskardin
- L Waqar
- M Waterfield
- P Weiser
- P Weiss
- E Wershba
- A Wise
- The University of Edinburgh, Edinburgh, UK
- L Woolnough
- E Wu
- 2University of Pennsylvania, Philadelphia, USA
- A Yalcindag
- M Yee
- Division of Diabetes, Endocrinology and Metabolism, Department of Medicine, Imperial College, London, UK
- E Yen
- Department of Gastroenterology, NorthShore Hospital, University of Chicago, Chicago, Illinois, USA
- R Yeung
- K Yomogida
- Q Yu
- Departments of Neurological Surgery and Neurology, Columbia College of Physicians and Surgeons, New York, NY, USA
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- A Zartoshti
- R Zeft
- A Zhu
- C Zic
- DOI
- https://doi.org/10.1136/lupus-2021-000494
- Journal volume & issue
-
Vol. 8,
no. 1
Abstract
Introduction Direct-to-family clinical trials efficiently provide data while reducing the participation burden for children and their families. Although these trials can offer significant advantages over traditional clinical trials, the process of designing and implementing direct-to-family studies is poorly defined, especially in children with rheumatic disease. This paper provides lessons learnt from the design and implementation of a self-controlled, direct-to-family pilot trial aimed to evaluate the effects of a medication management device on adherence to hydroxychloroquine in paediatric SLE.Methods Several design features accommodate a direct-to-family approach. Participants meeting eligibility criteria from across the USA were identified a priori through a disease registry, and all outcome data are collected remotely. The primary outcome (medication adherence) is evaluated using electronic medication event-monitoring, plasma drug levels, patient questionnaires and pill counts. Secondary and exploratory endpoints include (1) lupus disease activity measured by a remote SLE Disease Activity Index examination and the Systemic Lupus Activity Questionnaire; and (2) hydroxychloroquine pharmacokinetics and pharmacodynamics. Recruitment of the initial target of 20 participants was achieved within 10 days. Due to initial recruitment success, enrolment was increased to 26 participants. Additional participants who were interested were placed on a waiting list in case of dropouts during the study.Discussion and dissemination Direct-to-family trials offer several advantages but present unique challenges. Lessons learnt from the protocol development, design, and implementation of this trial will inform future direct-to-family trials for children and adults with rheumatic diseases. Additionally, the data collected remotely in this trial will provide critical information regarding the accuracy of teleresearch in lupus, the impact of adherence to hydroxychloroquine on disease activity and a pharmacokinetic analysis to inform paediatric-specific dosing of hydroxychloroquine.Trial registration number ClinicalTrials.gov Registry (NCT04358302).