Clinical complications in severe pediatric sickle cell disease and the impact of hydroxyurea

Tripathi, Avnish; Jerrell, Jeanette M.; Stallworth, James

doi:10.1002/pbc.22822

Cited by 23 publications

(18 citation statements)

References 25 publications

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“…Because of the benefit of hydroxyurea in decreasing acute sickle cell-related complications, clinicians should consider shifting their practice to prescribe hydroxyurea therapy for all very young children with SCA, rather than treating only those most severely affected. 38 BLOOD, 22 NOVEMBER 2012 ⅐ VOLUME 120, NUMBER 22 For personal use only. on May 10, 2018. by guest www.bloodjournal.org From…”

Section: Discussionmentioning

confidence: 99%

Impact of hydroxyurea on clinical events in the BABY HUG trial

Thornburg

Files

Luo

et al. 2012

Blood

215

185

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The Pediatric Hydroxyurea Phase 3 Clinical Trial (BABY HUG) was a phase 3 multicenter, randomized, double-blind, placebocontrolled clinical trial of hydroxyurea in infants (beginning at 9-18 months of age) with sickle cell anemia. An important secondary objective of this study was to compare clinical events between the hydroxyurea and placebo groups. One hundred and ninetythree subjects were randomized to hy- Continuing Medical Education onlineThis activity has been planned and implemented in accordance with the Essential Areas and policies of the Accreditation Council for Continuing Medical Education through the joint sponsorship of Medscape, LLC and the American Society of Hematology. Medscape, LLC is accredited by the ACCME to provide continuing medical education for physicians. Medscape, LLC designates this Journal-based CME activity for a maximum of 1.0 AMA PRA Category 1 Credit(s) ™ . Physicians should claim only the credit commensurate with the extent of their participationin the activity. All other clinicians completing this activity will be issued a certificate of participation. To participate in this journal CME activity: (1) review the learning objectives and author disclosures; (2) study the education content; (3) take the post-test with a 70% minimum passing score and complete the evaluation at http://www.medscape.org/journal/blood; and (4) view/print certificate. For CME questions, see page 4448. Disclosures The authors, the Associate Editor Narla Mohandas, and CME questions author Laurie Barclay, freelance writer and reviewer, Medscape, LLC, declare no competing financial interests. Learning objectivesUpon completion of this activity, participants will be able to:1. Describe the effect of hydroxyurea on rates of sickle cell complications for infants with sickle cell anemia (SCA) on the basis of a phase 3, multicenter, randomized trial.2. Describe the effect of hydroxyurea on rates of hospitalizations and transfusions for infants with SCA on the basis of a phase 3, multicenter, randomized trial.3. Describe the safety of hydroxyurea for infants with SCA on the basis of a phase 3, multicenter, randomized trial.

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Section: Discussionmentioning

confidence: 99%

Impact of hydroxyurea on clinical events in the BABY HUG trial

Thornburg

Files

Luo

et al. 2012

Blood

215

185

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“…[6,14] Need for research on specific barriers to HU use has been highlighted. [15] Insights from prior research in other health conditions suggest barriers to medication use among chronically ill under-served populations generally involve provider-patient relationship dynamics [16–18].…”

Section: Introductionmentioning

confidence: 99%

Parental and other factors associated with hydroxyurea use for pediatric sickle cell disease

Oyeku

Driscoll

Cohen

et al. 2012

Pediatric Blood & Cancer

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Background Hydroxyurea (HU) is highly effective treatment for Sickle Cell Disease (SCD). While pediatric use of HU is accepted clinical practice, barriers to use may impede its potential benefit. Procedure A survey of parents of children ages 5–17 years with SCD was performed across five institutions to assess factors associated with HU use. Results Of the 173 parent responses, 65 (38%) had children currently taking HU. Among parents of children not taking HU, the most commonly cited reasons were that their hematology provider had not offered it, their child was not sufficiently symptomatic and concerns about potential side effects. Even parents of HU users reported widespread concern about effectiveness, long-term safety and off-label use. In bivariate analyses, children’s ages, parental demographics such as education level, or travel time to their hematology provider were not correlated with HU use. Bivariate analysis and multivariate logistic regression revealed three significant factors associated with current HU use: better parental knowledge about its major therapeutic effects (p<0.001), sickle genotype (p=0.005) and institution of clinical care (p=0.04). Conclusions Pervasive concerns about HU safety exist, even among parents of current users. Varying knowledge among parents appears to be independent of their demographics, and is associated with HU use. Inter-institutional variability in parental knowledge and drug uptake highlights potentially potent site-specific influences on likelihood of HU use. Overall, these survey data underscore the need for strategies to bolster parental understanding about benefits of HU and address concerns about its safety.

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“…Many complications may occur that involve different organs such as splenic sequestration, cerebrovascular accident, acute chest syndrome, pulmonary hypertension, priapism, cholelithiasis, bone infarctions, and retinopathy (National Institutes of Health [NIH], 2002;Pack-Mabien & Haynes, 2009). The recurrent vasoocclusion may damage organs (renal, lung, liver), delay puberty, and affect neurocognition (Rees, Williams, & Gladwin, 2010;Tripathi, Jerrell, & Stallworth, 2011). Some individuals with SCD begin having symptoms during the first year of life, presenting with dactylitis (hand-foot syndrome), febrile illness and infections, or aplastic crisis.…”

mentioning

confidence: 99%

Remote monitoring of pain and symptoms using wireless technology in children and adolescents with sickle cell disease

Jacob

Duran

Stinson

et al. 2012

Journal of the American Academy of Nurse Practitioners

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Purpose The purpose of this study was to examine 1) symptoms; 2) pain characteristics (intensity, location, quality); 3) pain medications and nonpharmacological strategies used for pain; 4) thoughts and feelings; and 5) health care visits. We also examined the relationship between pain and sleep. Data Sources Pain and symptoms were entered on an electronic e-Diary using a smartphone and were remotely monitored by an advanced practice registered nurse. Sixty-seven children and adolescents (10 to 17 years) reported mild to severe pain at home that did not require health care visits. Symptoms reported were: 1) general symptoms such as tiredness/fatigue (34.7%), headache (20.8%), yellowing of the eyes (28.4%); 2) respiratory symptoms such as sniffling (32.9%), coughing (19.1%), changes in breathing (10.0%); and 3) musculoskeletal symptoms such as stiffness in joints (15.8%). A significant negative correlation was found between pain and sleep (r = −0.387, p=0.024). Factors that predict pain included previous history of SCD related events, symptoms, and negative thoughts. Conclusion Pain and multiple symptoms entered on a web-based e-Diary were remotely monitored by an APRN and prompted communications, further evaluation, and recommendations. Implications for Practice Remote monitoring using wireless technology may facilitate timely management of pain and symptoms and minimize negative consequences in SCD.

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Clinical complications in severe pediatric sickle cell disease and the impact of hydroxyurea

Cited by 23 publications

References 25 publications

Impact of hydroxyurea on clinical events in the BABY HUG trial

Impact of hydroxyurea on clinical events in the BABY HUG trial

Parental and other factors associated with hydroxyurea use for pediatric sickle cell disease

Remote monitoring of pain and symptoms using wireless technology in children and adolescents with sickle cell disease

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