Effects of hydroxyurea treatment for patients with hemoglobin SC disease

Lori Luchtman-Jones*, Sara Pressel, Lee Hilliard, R. Clark Brown, Mary G. Smith, Alexis A. Thompson, Margaret T. Lee, Jennifer Rothman, Zora R. Rogers, William Owen, Hamayun Imran, Courtney Thornburg, Janet L. Kwiatkowski, Banu Aygun, Stephen Nelson, Carla Roberts, Cynthia Gauger, Connie Piccone, Theodosia Kalfa, Ofelia Alvarez & 3 others Kathryn Hassell, Barry R. Davis, Russell E. Ware

*Corresponding author for this work

Research output: Contribution to journalArticle

22 Citations (Scopus)

Abstract

Although hemoglobin SC (HbSC) disease is usually considered less severe than sickle cell anemia (SCA), which includes HbSS and HbS/β0-thalassemia genotypes, many patients with HbSC experience severe disease complications, including vaso-occlusive pain, acute chest syndrome, avascular necrosis, retinopathy, and poor quality of life. Fully 20 years after the clinical and laboratory efficacy of hydroxyurea was proven in adult SCA patients, the safety and utility of hydroxyurea treatment for HbSC patients remain unclear. Recent NHLBI evidence-based guidelines highlight this as a critical knowledge gap, noting HbSC accounts for ∼30% of sickle cell patients within the United States. To date, only 5 publications have reported short-term, incomplete, or conflicting laboratory and clinical outcomes of hydroxyurea treatment in a total of 71 adults and children with HbSC. We now report on a cohort of 133 adult and pediatric HbSC patients who received hydroxyurea, typically for recurrent vaso-occlusive pain. Hydroxyurea treatment was associated with a stable hemoglobin concentration; increased fetal hemoglobin (HbF) and mean corpuscular volume (MCV); and reduced white blood cell count (WBC), absolute neutrophil count (ANC), and absolute reticulocyte count (ARC). Reversible cytopenias occurred in 22% of patients, primarily neutropenia and thrombocytopenia. Painful events were reduced with hydroxyurea, more in patients >15 years old. These multicenter data support the safety and potentially salutary effects of hydroxyurea treatment for HbSC disease; however, a multicenter, placebo-controlled, Phase 3 clinical trial is needed to determine if hydroxyurea therapy has efficacy for patients with HbSC disease.

Original languageEnglish (US)
Pages (from-to)238-242
Number of pages5
JournalAmerican Journal of Hematology
Volume91
Issue number2
DOIs
StatePublished - Feb 1 2016

Fingerprint

Hemoglobin SC Disease
Hydroxyurea
Hemoglobins
Sickle Cell Anemia
Therapeutics
Acute Chest Syndrome
National Heart, Lung, and Blood Institute (U.S.)
Reticulocyte Count
Pain
Fetal Hemoglobin
Phase III Clinical Trials
Erythrocyte Indices
Thalassemia
Patient Safety
Neutropenia
Leukocyte Count
Thrombocytopenia
Publications
Neutrophils
Necrosis

ASJC Scopus subject areas

  • Hematology

Cite this

Luchtman-Jones, L., Pressel, S., Hilliard, L., Brown, R. C., Smith, M. G., Thompson, A. A., ... Ware, R. E. (2016). Effects of hydroxyurea treatment for patients with hemoglobin SC disease. American Journal of Hematology, 91(2), 238-242. https://doi.org/10.1002/ajh.24255
Luchtman-Jones, Lori ; Pressel, Sara ; Hilliard, Lee ; Brown, R. Clark ; Smith, Mary G. ; Thompson, Alexis A. ; Lee, Margaret T. ; Rothman, Jennifer ; Rogers, Zora R. ; Owen, William ; Imran, Hamayun ; Thornburg, Courtney ; Kwiatkowski, Janet L. ; Aygun, Banu ; Nelson, Stephen ; Roberts, Carla ; Gauger, Cynthia ; Piccone, Connie ; Kalfa, Theodosia ; Alvarez, Ofelia ; Hassell, Kathryn ; Davis, Barry R. ; Ware, Russell E. / Effects of hydroxyurea treatment for patients with hemoglobin SC disease. In: American Journal of Hematology. 2016 ; Vol. 91, No. 2. pp. 238-242.
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abstract = "Although hemoglobin SC (HbSC) disease is usually considered less severe than sickle cell anemia (SCA), which includes HbSS and HbS/β0-thalassemia genotypes, many patients with HbSC experience severe disease complications, including vaso-occlusive pain, acute chest syndrome, avascular necrosis, retinopathy, and poor quality of life. Fully 20 years after the clinical and laboratory efficacy of hydroxyurea was proven in adult SCA patients, the safety and utility of hydroxyurea treatment for HbSC patients remain unclear. Recent NHLBI evidence-based guidelines highlight this as a critical knowledge gap, noting HbSC accounts for ∼30{\%} of sickle cell patients within the United States. To date, only 5 publications have reported short-term, incomplete, or conflicting laboratory and clinical outcomes of hydroxyurea treatment in a total of 71 adults and children with HbSC. We now report on a cohort of 133 adult and pediatric HbSC patients who received hydroxyurea, typically for recurrent vaso-occlusive pain. Hydroxyurea treatment was associated with a stable hemoglobin concentration; increased fetal hemoglobin (HbF) and mean corpuscular volume (MCV); and reduced white blood cell count (WBC), absolute neutrophil count (ANC), and absolute reticulocyte count (ARC). Reversible cytopenias occurred in 22{\%} of patients, primarily neutropenia and thrombocytopenia. Painful events were reduced with hydroxyurea, more in patients >15 years old. These multicenter data support the safety and potentially salutary effects of hydroxyurea treatment for HbSC disease; however, a multicenter, placebo-controlled, Phase 3 clinical trial is needed to determine if hydroxyurea therapy has efficacy for patients with HbSC disease.",
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Luchtman-Jones, L, Pressel, S, Hilliard, L, Brown, RC, Smith, MG, Thompson, AA, Lee, MT, Rothman, J, Rogers, ZR, Owen, W, Imran, H, Thornburg, C, Kwiatkowski, JL, Aygun, B, Nelson, S, Roberts, C, Gauger, C, Piccone, C, Kalfa, T, Alvarez, O, Hassell, K, Davis, BR & Ware, RE 2016, 'Effects of hydroxyurea treatment for patients with hemoglobin SC disease', American Journal of Hematology, vol. 91, no. 2, pp. 238-242. https://doi.org/10.1002/ajh.24255

Effects of hydroxyurea treatment for patients with hemoglobin SC disease. / Luchtman-Jones, Lori; Pressel, Sara; Hilliard, Lee; Brown, R. Clark; Smith, Mary G.; Thompson, Alexis A.; Lee, Margaret T.; Rothman, Jennifer; Rogers, Zora R.; Owen, William; Imran, Hamayun; Thornburg, Courtney; Kwiatkowski, Janet L.; Aygun, Banu; Nelson, Stephen; Roberts, Carla; Gauger, Cynthia; Piccone, Connie; Kalfa, Theodosia; Alvarez, Ofelia; Hassell, Kathryn; Davis, Barry R.; Ware, Russell E.

In: American Journal of Hematology, Vol. 91, No. 2, 01.02.2016, p. 238-242.

Research output: Contribution to journalArticle

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AU - Luchtman-Jones, Lori

AU - Pressel, Sara

AU - Hilliard, Lee

AU - Brown, R. Clark

AU - Smith, Mary G.

AU - Thompson, Alexis A.

AU - Lee, Margaret T.

AU - Rothman, Jennifer

AU - Rogers, Zora R.

AU - Owen, William

AU - Imran, Hamayun

AU - Thornburg, Courtney

AU - Kwiatkowski, Janet L.

AU - Aygun, Banu

AU - Nelson, Stephen

AU - Roberts, Carla

AU - Gauger, Cynthia

AU - Piccone, Connie

AU - Kalfa, Theodosia

AU - Alvarez, Ofelia

AU - Hassell, Kathryn

AU - Davis, Barry R.

AU - Ware, Russell E.

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