Hydroxycarbamide versus chronic transfusion for maintenance of transcranial doppler flow velocities in children with sickle cell anaemia - TCD with Transfusions Changing to Hydroxyurea (TWiTCH): A multicentre, open-label, phase 3, non-inferiority trial

The Lancet

Volumn 387, Issue 10019, 2016, Pages 661-670

  Ware, Russell E ^a   Davis, Barry R ^b   Schultz, William H ^a   Brown, R Clark ^c   Aygun, Banu ^d   Sarnaik, Sharada ^e   Odame, Isaac ^f   Fuh, Beng ^g   George, Alex ^h   Owen, William ⁱ   Luchtman Jones, Lori ^j   Rogers, Zora R ^k   Hilliard, Lee ^l   Gauger, Cynthia ^m   Piccone, Connie ⁿ   Lee, Margaret T ^o   Kwiatkowski, Janet L ^p   Jackson, Sherron ^q   Miller, Scott T ^r   Roberts, Carla ^s   Heeney, Matthew M ^t   Kalfa, Theodosia A ^a   Nelson, Stephen ^u   Imran, Hamayun ^v   Nottage, Kerri ^w   Alvarez, Ofelia ^x   Rhodes, Melissa ^y   Thompson, Alexis A ^z   Rothman, Jennifer A ^aa   Helton, Kathleen J ^w   Roberts, Donna ^q   Coleman, Jamie ^w   Bonner, Melanie J ^aa   Kutlar, Abdullah ^ab   Patel, Niren ^ab   Wood, John ^ac   Piller, Linda ^b   Wei, Peng ^b   Luden, Judy ^q   Mortier, Nicole A ^a   Stuber, Susan E ^a   Luban, Naomi L C ^j   Cohen, Alan R ^p   Pressel, Sara ^b   Adams, Robert J ^q   more..

^a CINCINNATI CHILDREN'S HOSPITAL MEDICAL CENTER   (United States)

^b UNIVERSITY OF TEXAS SCHOOL OF PUBLIC HEALTH   (United States)

^c EMORY UNIVERSITY   (United States)

^d Northwell Health   (United States)

^e WAYNE STATE UNIVERSITY   (United States)

^f HOSPITAL FOR SICK CHILDREN   (Canada)

^g EAST CAROLINA UNIVERSITY   (United States)

^h BAYLOR COLLEGE OF MEDICINE   (United States)

ⁱ CHILDREN S HOSPITAL OF THE KING S DAUGHTERS   (United States)

^j CHILDREN'S NATIONAL MEDICAL CENTER   (United States)

^k UNIVERSITY OF TEXAS SOUTHWESTERN MEDICAL CENTER   (United States)

^l UNIVERSITY OF ALABAMA AT BIRMINGHAM   (United States)

^m NEMOURS CHILDREN S CLINIC   (United States)

ⁿ CASE WESTERN RESERVE UNIVERSITY   (United States)

^o Och Spine at New York Presbyterian Hospitals   (United States)

^p CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

^q MEDICAL UNIVERSITY OF SOUTH CAROLINA   (United States)

^r SUNY DOWNSTATE MEDICAL CENTER   (United States)

^s UNIVERSITY OF SOUTH CAROLINA   (United States)

^t BOSTON CHILDREN S HOSPITAL   (United States)

^u Children's Hospitals   (United States)

^v UNIVERSITY OF SOUTH ALABAMA   (United States)

^w ST JUDE CHILDREN S RESEARCH HOSPITAL   (United States)

^x UNIVERSITY OF MIAMI   (United States)

^y UNIVERSITY OF MISSISSIPPI   (United States)

^z CHILDREN'S MEMORIAL HOSPITAL   (United States)

^aa DUKE UNIVERSITY MEDICAL CENTER   (United States)

^ab AUGUSTA UNIVERSITY   (United States)

^ac CHILDREN S HOSPITAL LOS ANGELES   (United States)

more..

Author keywords

[No Author keywords available]

Indexed keywords

HEMOGLOBIN S; HYDROXYUREA; ANTISICKLING AGENT;

ACUTE CHEST SYNDROME; ADOLESCENT; ARTICLE; BLOOD TRANSFUSION; BRAIN INFARCTION; CEREBROVASCULAR ACCIDENT; CHILD; CONTROLLED STUDY; DRUG DOSE ESCALATION; DRUG SAFETY; FEMALE; FEVER; FLOW RATE; HEPATOBILIARY DISEASE; HUMAN; INFECTION; INTENTION TO TREAT ANALYSIS; MAGNETIC RESONANCE ANGIOGRAPHY; MAJOR CLINICAL STUDY; MALE; MAXIMUM TOLERATED DOSE; MULTICENTER STUDY; NEUROLOGIC DISEASE; NUCLEAR MAGNETIC RESONANCE IMAGING; PAIN; PHASE 3 CLINICAL TRIAL; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL; SICKLE CELL ANEMIA; SPLENECTOMY; SPLENOMEGALY; TRANSCRANIAL DOPPLER; TRANSIENT ISCHEMIC ATTACK; TREATMENT DURATION; VASCULAR DISEASE; VASOOCCLUSIVE PAIN; ANEMIA, SICKLE CELL; BLOOD FLOW VELOCITY; BRAIN CIRCULATION; CLINICAL TRIAL; COMPARATIVE STUDY; DOPPLER ECHOGRAPHY; DRUG SUBSTITUTION; MULTIMODALITY CANCER THERAPY; PATHOPHYSIOLOGY; PHYSIOLOGY; PRESCHOOL CHILD; PROCEDURES; STROKE; TREATMENT OUTCOME;

ADOLESCENT; ANEMIA, SICKLE CELL; ANTISICKLING AGENTS; BLOOD FLOW VELOCITY; BLOOD TRANSFUSION; CEREBROVASCULAR CIRCULATION; CHILD; CHILD, PRESCHOOL; COMBINED MODALITY THERAPY; DRUG SUBSTITUTION; FEMALE; HUMANS; HYDROXYUREA; MALE; STROKE; TREATMENT OUTCOME; ULTRASONOGRAPHY, DOPPLER, TRANSCRANIAL;

EID: 84957891583     PISSN: 01406736     EISSN: 1474547X     Source Type: Journal    
DOI: 10.1016/S0140-6736(15)01041-7     Document Type: Article

References (30)

1. K Ohene-Frempong, SJ Weiner, LA Sleeper et al. Cerebrovascular accidents in sickle cell disease: rates and risk factors Blood 91 1998 288 294
2. ML Hulbert, RC McKinstry, JL Lacey et al. Silent cerebral infarcts occur despite regular blood transfusion therapy after first strokes in children with sickle cell disease Blood 117 2011 772 779
3. R Adams, V McKie, F Nichols et al. The use of transcranial ultrasonography to predict stroke in sickle cell disease N Engl J Med 326 1992 605 610
4. RJ Adams, VC McKie, L Hsu et al. Prevention of a first stroke by transfusion in children with sickle cell anemia and abnormal results on transcranial Doppler ultrasonography N Engl J Med 339 1998 5 11
5. RJ Adams for the STOP 2 Trial Investigators Discontinuing prophylactic transfusions used to prevent stroke in sickle cell disease N Engl J Med 353 2005 2769 2778
6. BP Yawn, GR Buchanan, AN Afenyi-Annan et al. Management of sickle cell disease. Summary of the 2014 evidence-based report by expert panel members JAMA 312 2014 1033 1048
7. F Bernaudin, S Verlhac, C Arnaud et al. Impact of early transcranial Doppler screening and intensive therapy on cerebral vasculopathy outcome in a newborn sickle cell anemia cohort Blood 117 2011 1130 1140
8. S Charache, ML Terrin, RD Moore et al. Effect of hydroxyurea on the frequency of painful crises in sickle cell anemia N Engl J Med 332 1995 1317 1322
9. WC Wang, RE Ware, ST Miller for the BABY HUG Investigators Hydroxycarbamide in very young children with sickle-cell anaemia: a multicentre, randomised, controlled trial (BABY HUG) Lancet 377 2011 1663 1672
10. RE Ware How I use hydroxyurea to treat young patients with sickle cell anemia Blood 115 2010 5300 5311
11. B Gulbis, D Haberman, D Dufour et al. Hydroxyurea for sickle cell disease in children and for prevention of cerebrovascular events: the Belgian experience Blood 105 2005 2685 2690
12. SA Zimmerman, WH Schultz, S Burgett, NA Mortier, RE Ware Hydroxyurea therapy lowers transcranial Doppler flow velocities in children with sickle cell anemia Blood 110 2007 1043 1047
13. KJ Helton, RJ Adams, KL Kesler et al. Magnetic resonance imaging/angiography and transcranial Doppler velocities in sickle cell anemia: results from the SWiTCH trial Blood 124 2014 891 898
14. MM Heeney, RE Ware Hydroxyurea for children with sickle cell disease Pediatr Clin North Am 55 2008 483 501
15. Ware RE. Optimizing hydroxyurea therapy for sickle cell anemia. Hematology Am Soc Hematol Educ Program (in press).
16. B Aygun, NA Mortier, K Kesler et al. Therapeutic phlebotomy is safe in children with sickle cell anemia and can be effective treatment for transfusional iron overload Br J Haematol 169 2015 262 266
17. DM Reboussin, DL DeMets, K Kyungmann, KK Lan Computations for group sequential boundaries using the Lan-DeMets spending function method Control Clin Trials 21 2000 190 207
18. DJ Brambilla, ST Miller, RJ Adams Intra-individual variation in blood flow velocities in cerebral arteries of children with sickle cell disease Pediatr Blood Cancer 49 2007 318 322
19. CB Almeida, LE Souza, FC Leonardo et al. Acute hemolytic vascular inflammatory processes are prevented by nitric oxide replacement or a single dose of hydroxyurea Blood 126 2015 711 720
20. JL Raphael, PB Shetty, H Lui, DH Mahoney, BU Mueller A critical assessment of transcranial Doppler screening rates in a large pediatric sickle cell center: opportunities to improve healthcare quality Pediatr Blood Cancer 51 2008 647 651
21. SL Reeves, HJ Fullerton, KJ Dombkowski, ML Boulton, TM Braun, LD Lisabeth Physician attitude, awareness, and knowledge regarding guidelines for transcranial Doppler screening in sickle cell disease Clin Pediatr 54 2015 336 345
22. B Aygun, M Wruck, WH Schultz et al. Chronic transfusion practices for prevention of primary stroke in children with sickle cell anemia and abnormal TCD velocities Am J Hematol 87 2012 428 430
23. MR DeBaun, M Gordon, RC McKinstry et al. Controlled trial of transfusions for silent cerebral infarcts in sickle cell anemia N Engl J Med 371 2014 699 710
24. JL Kwiatkowski, E Yim, S Miller, RJ Adams for the STOP2 Study Investigators Effect of transfusion therapy on transcranial Doppler ultrasonography velocities in children with sickle cell disease Pediatr Blood Cancer 56 2011 777 782
25. RE Ware, WH Schultz, N Yovetich et al. Stroke With Transfusions Changing to Hydroxyurea (SWiTCH): A Phase III randomized clinical trial for treatment of children with sickle cell anemia, stroke, and iron overload Pediatr Blood Cancer 57 2011 1011 1017
26. RE Ware, SA Zimmerman, PB Sylvestre et al. Prevention of secondary stroke and resolution of transfusional iron overload in children with sickle cell anemia using hydroxyurea and phlebotomy J Pediatr 145 2004 346 352
27. A Greenway, RE Ware, CD Thornburg Long-term results using hydroxyurea/phlebotomy for reducing secondary stroke risk in children with sickle cell anemia and iron overload Am J Hematol 86 2011 357 361
28. JM Flanagan, DM Frohlich, TA Howard et al. Genetic predictors for stroke in children with sickle cell anemia Blood 117 2011 6681 6684
29. JM Flanagan, VA Sheehan, H Linder et al. Genetic mapping and exome sequencing identify 2 mutations associated with stroke protection in pediatric patients with sickle cell anemia Blood 121 2013 3237 3245
30. FD Armstrong, TD Elkin, RC Brown for the BABY HUG Investigators Developmental function in toddlers with sickle cell anemia Pediatrics 131 2013 e406 e414