Hydroxyurea Therapy for Children With Sickle Cell Anemia in Sub-Saharan Africa: Rationale and Design of the REACH Trial

For the REACH Investigators

Research output: Contribution to journalArticle

13 Citations (Scopus)

Abstract

Background: Sickle cell anemia (SCA) is an inherited hematological disorder that causes a large but neglected global health burden, particularly in Africa. Hydroxyurea represents the only available disease-modifying therapy for SCA, and has proven safety and efficacy in high-resource countries. In sub-Saharan Africa, there is minimal use of hydroxyurea, due to lack of data, absence of evidence-based guidelines, and inexperience among healthcare providers. Procedure: A partnership was established between investigators in North America and sub-Saharan Africa, to develop a prospective multicenter research protocol designed to provide data on the safety, feasibility, and benefits of hydroxyurea for children with SCA. Results: The Realizing Effectiveness Across Continents with Hydroxyurea (REACH, ClinicalTrials.gov NCT01966731) trial is a prospective, phase I/II open-label dose escalation study of hydroxyurea that will treat a total of 600 children age 1-10 years with SCA: 150 at each of four different clinical sites within sub-Saharan Africa (Angola, Democratic Republic of Congo, Kenya, and Uganda). The primary study endpoint will be severe hematological toxicities that occur during the fixed-dose treatment phase. REACH has an adaptive statistical design that allows for careful assessment of toxicities to accurately identify a safe hydroxyurea dose. Conclusions: REACH will provide data that address critical gaps in knowledge for the treatment of SCA in sub-Saharan Africa. By developing local expertise with the use of hydroxyurea and helping to establish treatment guidelines, the REACH trial results will have the potential to transform care for children with SCA in Africa.

Original languageEnglish (US)
Pages (from-to)98-104
Number of pages7
JournalPediatric Blood and Cancer
Volume63
Issue number1
DOIs
StatePublished - Jan 1 2016

Fingerprint

Hydroxyurea
Africa South of the Sahara
Sickle Cell Anemia
Therapeutics
Angola
Guidelines
Democratic Republic of the Congo
Safety
Uganda
Kenya
Child Care
North America
Health Personnel
Research Personnel
Research

Keywords

  • Africa
  • Hydroxyurea
  • Sickle cell anemia

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Hematology
  • Oncology

Cite this

Hydroxyurea Therapy for Children With Sickle Cell Anemia in Sub-Saharan Africa : Rationale and Design of the REACH Trial. / For the REACH Investigators.

In: Pediatric Blood and Cancer, Vol. 63, No. 1, 01.01.2016, p. 98-104.

Research output: Contribution to journalArticle

@article{083f4f329122442692053b233003f3b9,
title = "Hydroxyurea Therapy for Children With Sickle Cell Anemia in Sub-Saharan Africa: Rationale and Design of the REACH Trial",
abstract = "Background: Sickle cell anemia (SCA) is an inherited hematological disorder that causes a large but neglected global health burden, particularly in Africa. Hydroxyurea represents the only available disease-modifying therapy for SCA, and has proven safety and efficacy in high-resource countries. In sub-Saharan Africa, there is minimal use of hydroxyurea, due to lack of data, absence of evidence-based guidelines, and inexperience among healthcare providers. Procedure: A partnership was established between investigators in North America and sub-Saharan Africa, to develop a prospective multicenter research protocol designed to provide data on the safety, feasibility, and benefits of hydroxyurea for children with SCA. Results: The Realizing Effectiveness Across Continents with Hydroxyurea (REACH, ClinicalTrials.gov NCT01966731) trial is a prospective, phase I/II open-label dose escalation study of hydroxyurea that will treat a total of 600 children age 1-10 years with SCA: 150 at each of four different clinical sites within sub-Saharan Africa (Angola, Democratic Republic of Congo, Kenya, and Uganda). The primary study endpoint will be severe hematological toxicities that occur during the fixed-dose treatment phase. REACH has an adaptive statistical design that allows for careful assessment of toxicities to accurately identify a safe hydroxyurea dose. Conclusions: REACH will provide data that address critical gaps in knowledge for the treatment of SCA in sub-Saharan Africa. By developing local expertise with the use of hydroxyurea and helping to establish treatment guidelines, the REACH trial results will have the potential to transform care for children with SCA in Africa.",
keywords = "Africa, Hydroxyurea, Sickle cell anemia",
author = "{For the REACH Investigators} and McGann, {Patrick T.} and L{\'e}on Tshilolo and Brigida Santos and Tomlinson, {George A.} and Susan Stuber and Teresa Latham and Banu Aygun and Obaro, {Stephen K} and Peter Olupot-Olupot and Williams, {Thomas N.} and Isaac Odame and Ware, {Russell E.}",
year = "2016",
month = "1",
day = "1",
doi = "10.1002/pbc.25705",
language = "English (US)",
volume = "63",
pages = "98--104",
journal = "Pediatric Blood and Cancer",
issn = "1545-5009",
publisher = "Wiley-Liss Inc.",
number = "1",

}

TY - JOUR

T1 - Hydroxyurea Therapy for Children With Sickle Cell Anemia in Sub-Saharan Africa

T2 - Rationale and Design of the REACH Trial

AU - For the REACH Investigators

AU - McGann, Patrick T.

AU - Tshilolo, Léon

AU - Santos, Brigida

AU - Tomlinson, George A.

AU - Stuber, Susan

AU - Latham, Teresa

AU - Aygun, Banu

AU - Obaro, Stephen K

AU - Olupot-Olupot, Peter

AU - Williams, Thomas N.

AU - Odame, Isaac

AU - Ware, Russell E.

PY - 2016/1/1

Y1 - 2016/1/1

N2 - Background: Sickle cell anemia (SCA) is an inherited hematological disorder that causes a large but neglected global health burden, particularly in Africa. Hydroxyurea represents the only available disease-modifying therapy for SCA, and has proven safety and efficacy in high-resource countries. In sub-Saharan Africa, there is minimal use of hydroxyurea, due to lack of data, absence of evidence-based guidelines, and inexperience among healthcare providers. Procedure: A partnership was established between investigators in North America and sub-Saharan Africa, to develop a prospective multicenter research protocol designed to provide data on the safety, feasibility, and benefits of hydroxyurea for children with SCA. Results: The Realizing Effectiveness Across Continents with Hydroxyurea (REACH, ClinicalTrials.gov NCT01966731) trial is a prospective, phase I/II open-label dose escalation study of hydroxyurea that will treat a total of 600 children age 1-10 years with SCA: 150 at each of four different clinical sites within sub-Saharan Africa (Angola, Democratic Republic of Congo, Kenya, and Uganda). The primary study endpoint will be severe hematological toxicities that occur during the fixed-dose treatment phase. REACH has an adaptive statistical design that allows for careful assessment of toxicities to accurately identify a safe hydroxyurea dose. Conclusions: REACH will provide data that address critical gaps in knowledge for the treatment of SCA in sub-Saharan Africa. By developing local expertise with the use of hydroxyurea and helping to establish treatment guidelines, the REACH trial results will have the potential to transform care for children with SCA in Africa.

AB - Background: Sickle cell anemia (SCA) is an inherited hematological disorder that causes a large but neglected global health burden, particularly in Africa. Hydroxyurea represents the only available disease-modifying therapy for SCA, and has proven safety and efficacy in high-resource countries. In sub-Saharan Africa, there is minimal use of hydroxyurea, due to lack of data, absence of evidence-based guidelines, and inexperience among healthcare providers. Procedure: A partnership was established between investigators in North America and sub-Saharan Africa, to develop a prospective multicenter research protocol designed to provide data on the safety, feasibility, and benefits of hydroxyurea for children with SCA. Results: The Realizing Effectiveness Across Continents with Hydroxyurea (REACH, ClinicalTrials.gov NCT01966731) trial is a prospective, phase I/II open-label dose escalation study of hydroxyurea that will treat a total of 600 children age 1-10 years with SCA: 150 at each of four different clinical sites within sub-Saharan Africa (Angola, Democratic Republic of Congo, Kenya, and Uganda). The primary study endpoint will be severe hematological toxicities that occur during the fixed-dose treatment phase. REACH has an adaptive statistical design that allows for careful assessment of toxicities to accurately identify a safe hydroxyurea dose. Conclusions: REACH will provide data that address critical gaps in knowledge for the treatment of SCA in sub-Saharan Africa. By developing local expertise with the use of hydroxyurea and helping to establish treatment guidelines, the REACH trial results will have the potential to transform care for children with SCA in Africa.

KW - Africa

KW - Hydroxyurea

KW - Sickle cell anemia

UR - http://www.scopus.com/inward/record.url?scp=84983095657&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=84983095657&partnerID=8YFLogxK

U2 - 10.1002/pbc.25705

DO - 10.1002/pbc.25705

M3 - Article

C2 - 26275071

AN - SCOPUS:84983095657

VL - 63

SP - 98

EP - 104

JO - Pediatric Blood and Cancer

JF - Pediatric Blood and Cancer

SN - 1545-5009

IS - 1

ER -