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Hydroxyurea for Children and Adults with Hemoglobin SC Disease

Author/s: 
Yvonne A Dei-Adomakoh, Catherine I Segbefia, Teresa S Latham, Adam C Lane

Background: Hemoglobin SC (HbSC) is a common sickle hemoglobinopathy that causes acute complications, chronic organ damage, and early death with no established disease-modifying treatment. In this trial, we examined the safety and efficacy of hydroxyurea treatment in patients with HbSC.

Methods: Prospective Identification of Variables as Outcomes for Treatment (PIVOT) was a double-blind, randomized, placebo-controlled, non-inferiority phase 2 trial in which we assigned children and adults with HbSC in Ghana to 12 months of hydroxyurea or placebo. The primary end point was hematologic dose-limiting toxicities (DLTs), including cytopenias or elevated hemoglobin levels during 12 months of blinded treatment. Clinical end points included vaso-occlusive pain events, acute chest syndrome, hospitalizations, transfusions, and malaria. Quality-of-life measures, organ function assessments, and rheological measurements were also collected.

Results: Of the 243 enrolled patients (118 female), 212 eligible participants initiated blinded treatment at 20.0±5.0 mg/kg/day. DLTs occurred in more participants on hydroxyurea (33%) than the placebo (11%), with a difference of 22 percentage points (95% confidence interval [CI],11 to 34 percentage points), which exceeded the predefined 15 percentage point noninferiority margin. Elevated levels of hemoglobin occurred in 12 participants on hydroxyurea and 10 on the placebo. Hydroxyurea treatment was associated with 57.0 versus 149.6 vaso-occlusive pain events per 100 person-years (incidence rate ratio [IRR] 0.38; 95% CI, 0.28 to 0.52), and 12.9 versus 30.6 hospitalizations per 100 person-years (IRR 0.42; 95% CI, 0.22 to 0.81). A composite of acute sickle-related events occurred in 37 participants on hydroxyurea versus 69 participants on placebo (IRR 0.39; (95% CI, 0.26 to 0.59), a difference observed in both children and adults.

Conclusions: The PIVOT trial did not meet its primary end point. Hydroxyurea at 20 mg/kg in patients with HbSC was associated with more hematologic DLTs than placebo, but most were mild and transient. Hydroxyurea was associated with less vaso-occlusive pain and fewer sickle-related events in both children and adults; a new trial will need to be done to establish the efficacy of this approach. (Funded by Theravia; Pan-African Clinical Trials Registry number, PACTR 202108893981080).

Keywords 
hydroxyurea Hgb SC

Use of Recommended Health Care Measures to Prevent Selected Complications of Sickle Cell Anemia in Children and Adolescents — Selected U.S. States, 2019

Author/s: 
Schieve, L. A., Simmons, G. M., Payne, A. B., Abe, K., Hsu, L. L., Hulihan, M., Pope, S., Rhie, S., Dupervil, B., Hooper, W. C.

Introduction: Sickle cell disease (SCD), a group of inherited blood cell disorders that primarily affects Black or African American persons, is associated with severe complications and a >20-year reduction in life expectancy. In 2014, an expert panel convened by the National Heart, Lung, and Blood Institute issued recommendations to prevent or reduce complications in children and adolescents with the most severe SCD subtypes, known as sickle cell anemia (SCA); recommendations included 1) annual screening of children and adolescents aged 2–16 years with transcranial Doppler (TCD) ultrasound to identify those at risk for stroke and 2) offering hydroxyurea therapy to children and adolescents aged ≥9 months to reduce the risk for several life-threatening complications.

Methods: Data from the IBM MarketScan Multi-State Medicaid Database were analyzed. TCD screening and hydroxyurea use were examined for 3,352 children and adolescents with SCA aged 2–16 years and continuously enrolled in Medicaid during 2019. Percentage change during 2014–2019 and variation by health subgroups were assessed. Analyses were stratified by age.

Results: During 2014–2019, TCD screening increased 27% among children and adolescents aged 10–16 years; hydroxyurea use increased 27% among children aged 2–9 years and 23% among children and adolescents aged 10–16 years. However, in 2019, only 47% and 38% of children and adolescents aged 2–9 and 10–16 years, respectively, had received TCD screening and 38% and 53% of children and adolescents aged 2–9 years and 10–16 years, respectively, used hydroxyurea. For both prevention strategies, usage was highest among children and adolescents with high levels of health care utilization and evidence of previous complications indicative of severe disease.

Conclusion and Implications for Public Health Practice: Despite increases since 2014, TCD screening and hydroxyurea use remain low among children and adolescents with SCA. Health care providers should implement quality care strategies within their clinics and partner with patients, families, and community-based organizations to address barriers to delivering and receiving recommended care.

Keywords 
health personnel African Americans medicaid child blood cells

Coronavirus Disease 2019 (COVID-19): People with Certain Medical Conditions

Author/s: 
Centers for Disease Control and Prevention (CDC)

Revisions were made on November 2, 2020 to reflect recent data supporting increased risk of severe illness during pregnancy from the virus that causes COVID-19. Revisions also include addition of sickle cell disease and chronic kidney disease to the conditions that might increase the risk of severe illness among children.

We are learning more about COVID-19 every day. The below list of underlying medical conditions is not exhaustive and only includes conditions with sufficient evidence to draw conclusions; it is a living document that may be updated at any time, subject to potentially rapid change as the science evolves. This list is meant to inform clinicians to help them provide the best care possible for patients, and to inform individuals as to what their level of risk may be so they can make individual decisions about illness prevention. Notably, the list may not include every condition that might increase one’s risk for developing severe illness from COVID-19, such as those for which evidence may be limited or nonexistent (e.g., rare conditions). Individuals with any underlying condition (including those conditions that are NOT on the current list) should consult with their healthcare providers about personal risk factors and circumstances to determine whether extra precautions are warranted.

Keywords 
COVID-19 coronavirus underlying medical conditions high risk patients

The Many Faces of Cobalamin (Vitamin B12) Deficiency

Author/s: 
Wolffenbuttel, Bruce, Wouters, Hanneke J.C.M., Heiner-Fokkema, Rebecca, van der Klauw, Melanie M.

Although cobalamin (vitamin B12) deficiency was described over a century ago, it is still difficult toestablish the correct diagnosis and prescribe the right treatment. Symptoms related to vitamin B12 deficiency may be diverse and vary from neurologic to psychiatric. A number of individuals with vitamin B12 deficiency may present with the classic megaloblastic anemia.
In clinical practice, many cases of vitamin B12 deficiency are overlooked or sometimes even misdiagnosed. In this review, we describe the heterogeneous disease spectrum of patients with vitamin B12 deficiency in whom the diagnosis was either based on low serum B12 levels, elevated biomarkers like methylmalonic acid and/or homocysteine, or the improvement of clinical symptoms after the institution of parenteral vitamin B12 therapy. We discuss the possible clinical signs and symptoms of patients with B12 deficiency and the various pitfalls of diagnosis and treatment.

Keywords 
Vitamin B 12 Biomarkers diagnosis

The Many Faces of Cobalamin (Vitamin B12) Deficiency

Author/s: 
Wolffenbuttel, Bruce, Wouters, Hanneke J.C.M., Heiner-Fokkema, Rebecca, van der Klauw, Melanie M.

Although cobalamin (vitamin B12) deficiency was described over a century ago, it is still difficult toestablish the correct diagnosis and prescribe the right treatment. Symptoms related to vitamin B12 deficiency may be diverse and vary from neurologic to psychiatric. A number of individuals with vitamin B12 deficiency may present with the classic megaloblastic anemia.
In clinical practice, many cases of vitamin B12 deficiency are overlooked or sometimes even misdiagnosed. In this review, we describe the heterogeneous disease spectrum of patients with vitamin B12 deficiency in whom the diagnosis was either based on low serum B12 levels, elevated biomarkers like methylmalonic acid and/or homocysteine, or the improvement of clinical symptoms after the institution of parenteral vitamin B12 therapy. We discuss the possible clinical signs and symptoms of patients with B12 deficiency and the various pitfalls of diagnosis and treatment.

Keywords 
Vitamin B 12 Biomarkers diagnosis
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