Real-world experience using hydroxyurea in children with sickle cell disease in Lilongwe, Malawi

Tisungane Mvalo, Hillary M. Topazian, Portia Kamthunzi, Jane S. Chen, Isobel Kambalame, Pilirani Mafunga, Noel Mumba, Msandeni Chiume, Khadija Paseli, Gerald Tegha, Wiza Kumwenda, J. Brett Heimlich, Graham Ellis, Nigel Key, Satish Gopal, Irving Hoffman, Kenneth Ataga, Kate D. Westmoreland

Research output: Contribution to journalArticle

Abstract

Introduction: Sickle cell disease (SCD) is among the most common inherited hematologic diseases in sub-Saharan Africa (SSA). Historically, hydroxyurea administration in SSA has been restricted due to limited region-specific evidence for safety and efficacy. Methods: We conducted a prospective observational cohort study of pediatric patients with SCD in Malawi. From January 2015 to November 2017, hydroxyurea at doses of 10–20 mg/kg/day was administered to children with clinically severe disease (targeted use policy). From December 2017 to July 2018, hydroxyurea was prescribed to all patients (universal use policy). Results: Of 187 patients with SCD, seven (3.7%) died and 23 (12.3%) were lost to follow-up. The majority (135, 72.2%) were prescribed hydroxyurea, 59 (43.7%) under the targeted use policy and 76 (56.3%) under the universal use policy. There were no documented severe toxicities. Under the targeted use policy, children with SCD demonstrated absolute decreases in the rates of hospitalization (−4.1 per 1000 person-days; −7.2, −1.0; P =.004), fevers (−4.2 per 1000 person-days; −7.2, −1.1; P =.002), transfusions (−2.3 per 1000 person-days; 95% confidence interval: −4.9, 0.3; P =.06), and annual school absenteeism (−51.2 per person-year; −60.1, −42.3; P <.0001) within 6 months of hydroxyurea commencement. Conclusion: We successfully implemented universal administration of hydroxyurea to children with SCD at a tertiary hospital in Malawi. Similar to recently reported trials, hydroxyurea was safe and effective during routine programmatic experience, with clinical benefits particularly among high-risk children. This highlights the importance of continued widespread scale-up of hydroxyurea within SCD programs across SSA.

Original languageEnglish (US)
Article numbere27954
JournalPediatric Blood and Cancer
DOIs
StateAccepted/In press - Jan 1 2019

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Malawi
Hydroxyurea
Africa South of the Sahara
Sickle Cell Anemia
Pediatrics
Absenteeism
Hematologic Diseases
Lost to Follow-Up
Tertiary Care Centers
Observational Studies
Hospitalization
Cohort Studies
Fever
Confidence Intervals
Safety

All Science Journal Classification (ASJC) codes

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

Cite this

Mvalo, T., Topazian, H. M., Kamthunzi, P., Chen, J. S., Kambalame, I., Mafunga, P., ... Westmoreland, K. D. (Accepted/In press). Real-world experience using hydroxyurea in children with sickle cell disease in Lilongwe, Malawi. Pediatric Blood and Cancer, [e27954]. https://doi.org/10.1002/pbc.27954

Real-world experience using hydroxyurea in children with sickle cell disease in Lilongwe, Malawi. / Mvalo, Tisungane; Topazian, Hillary M.; Kamthunzi, Portia; Chen, Jane S.; Kambalame, Isobel; Mafunga, Pilirani; Mumba, Noel; Chiume, Msandeni; Paseli, Khadija; Tegha, Gerald; Kumwenda, Wiza; Heimlich, J. Brett; Ellis, Graham; Key, Nigel; Gopal, Satish; Hoffman, Irving; Ataga, Kenneth; Westmoreland, Kate D.

In: Pediatric Blood and Cancer, 01.01.2019.

Research output: Contribution to journalArticle

Mvalo, T, Topazian, HM, Kamthunzi, P, Chen, JS, Kambalame, I, Mafunga, P, Mumba, N, Chiume, M, Paseli, K, Tegha, G, Kumwenda, W, Heimlich, JB, Ellis, G, Key, N, Gopal, S, Hoffman, I, Ataga, K & Westmoreland, KD 2019, 'Real-world experience using hydroxyurea in children with sickle cell disease in Lilongwe, Malawi', Pediatric Blood and Cancer. https://doi.org/10.1002/pbc.27954
Mvalo, Tisungane ; Topazian, Hillary M. ; Kamthunzi, Portia ; Chen, Jane S. ; Kambalame, Isobel ; Mafunga, Pilirani ; Mumba, Noel ; Chiume, Msandeni ; Paseli, Khadija ; Tegha, Gerald ; Kumwenda, Wiza ; Heimlich, J. Brett ; Ellis, Graham ; Key, Nigel ; Gopal, Satish ; Hoffman, Irving ; Ataga, Kenneth ; Westmoreland, Kate D. / Real-world experience using hydroxyurea in children with sickle cell disease in Lilongwe, Malawi. In: Pediatric Blood and Cancer. 2019.
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abstract = "Introduction: Sickle cell disease (SCD) is among the most common inherited hematologic diseases in sub-Saharan Africa (SSA). Historically, hydroxyurea administration in SSA has been restricted due to limited region-specific evidence for safety and efficacy. Methods: We conducted a prospective observational cohort study of pediatric patients with SCD in Malawi. From January 2015 to November 2017, hydroxyurea at doses of 10–20 mg/kg/day was administered to children with clinically severe disease (targeted use policy). From December 2017 to July 2018, hydroxyurea was prescribed to all patients (universal use policy). Results: Of 187 patients with SCD, seven (3.7{\%}) died and 23 (12.3{\%}) were lost to follow-up. The majority (135, 72.2{\%}) were prescribed hydroxyurea, 59 (43.7{\%}) under the targeted use policy and 76 (56.3{\%}) under the universal use policy. There were no documented severe toxicities. Under the targeted use policy, children with SCD demonstrated absolute decreases in the rates of hospitalization (−4.1 per 1000 person-days; −7.2, −1.0; P =.004), fevers (−4.2 per 1000 person-days; −7.2, −1.1; P =.002), transfusions (−2.3 per 1000 person-days; 95{\%} confidence interval: −4.9, 0.3; P =.06), and annual school absenteeism (−51.2 per person-year; −60.1, −42.3; P <.0001) within 6 months of hydroxyurea commencement. Conclusion: We successfully implemented universal administration of hydroxyurea to children with SCD at a tertiary hospital in Malawi. Similar to recently reported trials, hydroxyurea was safe and effective during routine programmatic experience, with clinical benefits particularly among high-risk children. This highlights the importance of continued widespread scale-up of hydroxyurea within SCD programs across SSA.",
author = "Tisungane Mvalo and Topazian, {Hillary M.} and Portia Kamthunzi and Chen, {Jane S.} and Isobel Kambalame and Pilirani Mafunga and Noel Mumba and Msandeni Chiume and Khadija Paseli and Gerald Tegha and Wiza Kumwenda and Heimlich, {J. Brett} and Graham Ellis and Nigel Key and Satish Gopal and Irving Hoffman and Kenneth Ataga and Westmoreland, {Kate D.}",
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T1 - Real-world experience using hydroxyurea in children with sickle cell disease in Lilongwe, Malawi

AU - Mvalo, Tisungane

AU - Topazian, Hillary M.

AU - Kamthunzi, Portia

AU - Chen, Jane S.

AU - Kambalame, Isobel

AU - Mafunga, Pilirani

AU - Mumba, Noel

AU - Chiume, Msandeni

AU - Paseli, Khadija

AU - Tegha, Gerald

AU - Kumwenda, Wiza

AU - Heimlich, J. Brett

AU - Ellis, Graham

AU - Key, Nigel

AU - Gopal, Satish

AU - Hoffman, Irving

AU - Ataga, Kenneth

AU - Westmoreland, Kate D.

PY - 2019/1/1

Y1 - 2019/1/1

N2 - Introduction: Sickle cell disease (SCD) is among the most common inherited hematologic diseases in sub-Saharan Africa (SSA). Historically, hydroxyurea administration in SSA has been restricted due to limited region-specific evidence for safety and efficacy. Methods: We conducted a prospective observational cohort study of pediatric patients with SCD in Malawi. From January 2015 to November 2017, hydroxyurea at doses of 10–20 mg/kg/day was administered to children with clinically severe disease (targeted use policy). From December 2017 to July 2018, hydroxyurea was prescribed to all patients (universal use policy). Results: Of 187 patients with SCD, seven (3.7%) died and 23 (12.3%) were lost to follow-up. The majority (135, 72.2%) were prescribed hydroxyurea, 59 (43.7%) under the targeted use policy and 76 (56.3%) under the universal use policy. There were no documented severe toxicities. Under the targeted use policy, children with SCD demonstrated absolute decreases in the rates of hospitalization (−4.1 per 1000 person-days; −7.2, −1.0; P =.004), fevers (−4.2 per 1000 person-days; −7.2, −1.1; P =.002), transfusions (−2.3 per 1000 person-days; 95% confidence interval: −4.9, 0.3; P =.06), and annual school absenteeism (−51.2 per person-year; −60.1, −42.3; P <.0001) within 6 months of hydroxyurea commencement. Conclusion: We successfully implemented universal administration of hydroxyurea to children with SCD at a tertiary hospital in Malawi. Similar to recently reported trials, hydroxyurea was safe and effective during routine programmatic experience, with clinical benefits particularly among high-risk children. This highlights the importance of continued widespread scale-up of hydroxyurea within SCD programs across SSA.

AB - Introduction: Sickle cell disease (SCD) is among the most common inherited hematologic diseases in sub-Saharan Africa (SSA). Historically, hydroxyurea administration in SSA has been restricted due to limited region-specific evidence for safety and efficacy. Methods: We conducted a prospective observational cohort study of pediatric patients with SCD in Malawi. From January 2015 to November 2017, hydroxyurea at doses of 10–20 mg/kg/day was administered to children with clinically severe disease (targeted use policy). From December 2017 to July 2018, hydroxyurea was prescribed to all patients (universal use policy). Results: Of 187 patients with SCD, seven (3.7%) died and 23 (12.3%) were lost to follow-up. The majority (135, 72.2%) were prescribed hydroxyurea, 59 (43.7%) under the targeted use policy and 76 (56.3%) under the universal use policy. There were no documented severe toxicities. Under the targeted use policy, children with SCD demonstrated absolute decreases in the rates of hospitalization (−4.1 per 1000 person-days; −7.2, −1.0; P =.004), fevers (−4.2 per 1000 person-days; −7.2, −1.1; P =.002), transfusions (−2.3 per 1000 person-days; 95% confidence interval: −4.9, 0.3; P =.06), and annual school absenteeism (−51.2 per person-year; −60.1, −42.3; P <.0001) within 6 months of hydroxyurea commencement. Conclusion: We successfully implemented universal administration of hydroxyurea to children with SCD at a tertiary hospital in Malawi. Similar to recently reported trials, hydroxyurea was safe and effective during routine programmatic experience, with clinical benefits particularly among high-risk children. This highlights the importance of continued widespread scale-up of hydroxyurea within SCD programs across SSA.

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