Newborn Experience in the Cooperative Study of Sickle Cell Disease

PEDIATRICS ◽  
1989 ◽  
Vol 83 (5) ◽  
pp. 827-829
Author(s):  
Frances M. Gill ◽  
Audrey Brown ◽  
Dianne Gallagher ◽  
Steven Diamond ◽  
Eleanor Goins ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Newborn Screening ◽  
Sickle Cell ◽  
Large Scale ◽  
Collaborative Study ◽  
Developmental State ◽  
Cooperative Study ◽  
Cell Disease ◽  
Blood Institute ◽  
Rural And Urban

In 1978, the National Heart, Lung, and Blood Institute of the NIH initiated a large-scale, multiinstitutional collaborative study to investigate the clinical course of sickle cell disease from birth to death. Twenty-three institutions participate in the cooperative study, of which 19 have pediatric components. Newborn screening is performed in 18 institutions. Centers are located along the east coast, and in the South, the Midwest, and California. Both rural and urban populations are represented in the study. This Cooperative Study of Sickle Cell Disease has been described in detail. MATERIALS AND METHODS Newborn screening began in late 1978, and patient entry began in 1979. Between late 1978 and August 1986, 633 infants were found to have a hemoglobin pattern of FS and 326 were found to have FSC. A major effort was made to enroll these infants with sickle cell disease in the cooperative study. Other infants whose diseases were not diagnosed by screening but referred to the centers were also enrolled. From these two sources a total of 600 infants less than 6 months of age had been enrolled in the study as of August 1986. Diagnoses were established by cellulose acetate and citrate agar gel electrophoreses or by isoelectric focusing studies. The children have been followed up regularly at the study centers. Visits are scheduled for the ages of 2, 4, 6, 9, 12, 15, 18, 21, and 24 months and twice yearly thereafter. During the visits an interval history is obtained, a complete physical examination is performed, and developmental state is assessed.

scholarly journals Integration of Mobile Health Into Sickle Cell Disease Care to Increase Hydroxyurea Utilization: Protocol for an Efficacy and Implementation Study

10.2196/16319 ◽  
2020 ◽  
Vol 9 (7) ◽  
pp. e16319
Author(s):  
Jane S Hankins ◽  
Nirmish Shah ◽  
Lisa DiMartino ◽  
Donald Brambilla ◽  
Maria E Fernandez ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Mobile Health ◽  
Self Efficacy ◽  
Large Scale ◽  
Cell Disease ◽  
Prescribing Behavior ◽  
Blood Institute ◽  
Mhealth Intervention ◽  
Knowledge Barriers

Background Hydroxyurea prevents disease complications among patients with sickle cell disease (SCD). Although its efficacy has been endorsed by the National Health Lung and Blood Institute evidence-based guidelines, its adoption is low, both by patients with SCD and providers. Mobile health (mHealth) apps provide benefits in improving medication adherence and self-efficacy among patients with chronic diseases and have facilitated prescription among medical providers. However, mHealth has not been systematically tested as a tool to increase hydroxyurea adherence nor has the combination of mHealth been assessed at both patient and provider levels to increase hydroxyurea utilization. Objective This study aims to increase hydroxyurea utilization through a combined two-level mHealth intervention for both patients with SCD and their providers with the goals of increasing adherence to hydroxyurea among patients and improve hydroxyurea prescribing behavior among providers. Methods We will test the efficacy of 2 mHealth interventions to increase both patient and provider utilization and knowledge of hydroxyurea in 8 clinical sites of the NHLBI-funded Sickle Cell Disease Implementation Consortium (SCDIC). The patient mHealth intervention, InCharge Health, includes multiple components that address memory, motivation, and knowledge barriers to hydroxyurea use. The provider mHealth intervention, Hydroxyurea Toolbox (HU Toolbox), addresses the clinical knowledge barriers in prescribing and monitoring hydroxyurea. The primary hypothesis is that among adolescents and adults with SCD, adherence to hydroxyurea, as measured by the proportion of days covered (the ratio of the number of days the patient is covered by the medication to the number of days in the treatment period), will increase by at least 20% after 24 weeks of receiving the InCharge Health app, compared with their adherence at baseline. As secondary objectives, we will (1) examine the change in health-related quality of life, acute disease complications, perceived health literacy, and perceived self-efficacy in taking hydroxyurea among patients who use InCharge Health and (2) examine potential increases in the awareness of hydroxyurea benefits and risks, appropriate prescribing, and perceived self-efficacy to correctly administer hydroxyurea therapy among SCD providers between baseline and 9 months of using the HU Toolbox app. We will measure the reach, adoption, implementation, and maintenance of both the InCharge Health and the HU Toolbox apps using the reach, effectiveness, adoption, implementation, and maintenance framework and qualitatively evaluate the implementation of both mHealth interventions. Results The study is currently enrolling study participants. Recruitment is anticipated to be completed by mid-2021. Conclusions If this two-level intervention, that is, the combined use of InCharge Health and HU Toolbox apps, demonstrates efficacy in increasing adherence to hydroxyurea and prescribing behavior in patients with SCD and their providers, respectively, both apps will be offered to other institutions outside the SCDIC through a future large-scale implementation-effectiveness study. Trial Registration ClinicalTrials.gov NCT04080167; https://clinicaltrials.gov/ct2/show/NCT04080167 International Registered Report Identifier (IRRID) DERR1-10.2196/16319

scholarly journals Integration of Mobile Health Into Sickle Cell Disease Care to Increase Hydroxyurea Utilization: Protocol for an Efficacy and Implementation Study (Preprint)

2019 ◽  
Author(s):  
Jane S Hankins ◽  
Nirmish Shah ◽  
Lisa DiMartino ◽  
Donald Brambilla ◽  
Maria E Fernandez ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Mobile Health ◽  
Self Efficacy ◽  
Large Scale ◽  
Cell Disease ◽  
Prescribing Behavior ◽  
Blood Institute ◽  
Mhealth Intervention ◽  
Knowledge Barriers

BACKGROUND Hydroxyurea prevents disease complications among patients with sickle cell disease (SCD). Although its efficacy has been endorsed by the National Health Lung and Blood Institute evidence-based guidelines, its adoption is low, both by patients with SCD and providers. Mobile health (mHealth) apps provide benefits in improving medication adherence and self-efficacy among patients with chronic diseases and have facilitated prescription among medical providers. However, mHealth has not been systematically tested as a tool to increase hydroxyurea adherence nor has the combination of mHealth been assessed at both patient and provider levels to increase hydroxyurea utilization. OBJECTIVE This study aims to increase hydroxyurea utilization through a combined two-level mHealth intervention for both patients with SCD and their providers with the goals of increasing adherence to hydroxyurea among patients and improve hydroxyurea prescribing behavior among providers. METHODS We will test the efficacy of 2 mHealth interventions to increase both patient and provider utilization and knowledge of hydroxyurea in 8 clinical sites of the NHLBI-funded Sickle Cell Disease Implementation Consortium (SCDIC). The patient mHealth intervention, <i>InCharge Health,</i> includes multiple components that address memory, motivation, and knowledge barriers to hydroxyurea use. The provider mHealth intervention, <i>Hydroxyurea Toolbox</i> (<i>HU Toolbox</i>), addresses the clinical knowledge barriers in prescribing and monitoring hydroxyurea. The primary hypothesis is that among adolescents and adults with SCD, adherence to hydroxyurea, as measured by the proportion of days covered (the ratio of the number of days the patient is covered by the medication to the number of days in the treatment period), will increase by at least 20% after 24 weeks of receiving the <i>InCharge Health</i> app, compared with their adherence at baseline. As secondary objectives, we will (1) examine the change in health-related quality of life, acute disease complications, perceived health literacy, and perceived self-efficacy in taking hydroxyurea among patients who use <i>InCharge Health</i> and (2) examine potential increases in the awareness of hydroxyurea benefits and risks, appropriate prescribing, and perceived self-efficacy to correctly administer hydroxyurea therapy among SCD providers between baseline and 9 months of using the <i>HU Toolbox</i> app. We will measure the reach, adoption, implementation, and maintenance of both the <i>InCharge Health</i> and the <i>HU Toolbox</i> apps using the reach, effectiveness, adoption, implementation, and maintenance framework and qualitatively evaluate the implementation of both mHealth interventions. RESULTS The study is currently enrolling study participants. Recruitment is anticipated to be completed by mid-2021. CONCLUSIONS If this two-level intervention, that is, the combined use of InCharge Health and HU Toolbox apps, demonstrates efficacy in increasing adherence to hydroxyurea and prescribing behavior in patients with SCD and their providers, respectively, both apps will be offered to other institutions outside the SCDIC through a future large-scale implementation-effectiveness study. CLINICALTRIAL ClinicalTrials.gov NCT04080167; https://clinicaltrials.gov/ct2/show/NCT04080167 INTERNATIONAL REGISTERED REPORT DERR1-10.2196/16319

Social Work Perspective

PEDIATRICS ◽  
1989 ◽  
Vol 83 (5) ◽  
pp. 903-905
Author(s):  
Sandra Hernandez
Keyword(s):  
New York ◽  
Chronic Illness ◽  
Sickle Cell Disease ◽  
Newborn Screening ◽  
Sickle Cell ◽  
New York State ◽  
Early Years ◽  
Cell Disease ◽  
York State ◽  
The Impact

The ultimate objective of newborn screening for sickle cell disease should be twofold. The first essential step is the identification of the infants at risk. This has been effectively done in New York state as of 1975 through the New York State Newborn Screening Program. However, identifying these children is not enough. Second is the much more complicated task of providing comprehensive follow-up care for families whose children are affected by the disease, including the much needed psychosocial services. This area continues to be sorely neglected. The increased risk of death due to overwhelming infection in the first 3 years of life for children with sickle cell disease has been noted in the literature. When there is no specialized care, 15% to 20% do not survive. Therefore, it is essential for knowledgeable staff to make contact and begin to develop a trusting relationship as soon as possible with parents of infants born with sickle cell disease. Prophylactic penicillin and pneumococcal vaccination can reduce mortality during the early years. Family involvement with a consistent, available team of health care providers is pivotal in understanding this chronic illness and coping effectively with this extraordinary stress. Our staff is available by telephone for consultations with patients or other medical staff during clinic and emergency room visits and hospitalizations. One element that is clear in our experience at the St Luke's-Roosevelt Hospital Sickle Cell Center in New York City is that adjustment to this chronic illness is a lifelong process. One or two counseling sessions at the time of diagnosis are not sufficient to enable families to fully understand the information given or to realize the impact of having a child with a chronic illness.

Perspective From the American Academy of Pediatrics

PEDIATRICS ◽  
1989 ◽  
Vol 83 (5) ◽  
pp. 913-914
Author(s):  
Neil A. Holtzman
Keyword(s):  
Sickle Cell Disease ◽  
American Academy ◽  
Sickle Cell ◽  
Second Phase ◽  
Pilot Program ◽  
Cooperative Study ◽  
Cell Disease ◽  
Pilot Studies ◽  
American Academy Of Pediatrics

Ten years ago, the initial report of the Committee on Genetics of the American Academy of Pediatrics was published, drawing heavily on the landmark report published 2 years earlier by the National Academy of Sciences. That report stated that new mass screening tests should not be implemented without pilot studies or facilities for follow-up. I would like to deal with the efficacy of screening as determined by pilot studies, the effectiveness of routine screening, and the importance of follow-up. The Cooperative Study of Sickle Cell Disease was essentially the first phase of a large pilot program that systematically demonstrated that sepsis, meningitis, and acute splenic sequestration occurred in young infants with sickle cell disease. Previous reports suggested, and the cooperative study corroborated, that without prior diagnosis many of these infants would die. The randomized trial of oral penicillin prophylaxis was the second phase of the pilot program, demonstrating that early treatment significantly reduced mortality. Thus, the results of this pilot program, conducted with support of the National Institutes of Health, documented that newborn screening for sickle cell disease satisfied a major criterion for screening: An intervention capable of reducing mortality would be efficacious if applied before the usual age of clinical diagnosis. The pilot program was conducted under ideal conditions. The findings are not enough to conclude that screening for sickle cell disease and other hemoglobinopathies will always be effective when performed routinely.

Newborn Screening for Sickle Cell Disease: Effect on Mortality

PEDIATRICS ◽  
1988 ◽  
Vol 81 (6) ◽  
pp. 749-755
Author(s):  
Elliott Vichinsky ◽  
Deborah Hurst ◽  
Ann Earles ◽  
Klara Kleman ◽  
Bertram Lubin
Keyword(s):  
Sickle Cell Disease ◽  
Mortality Rate ◽  
Newborn Screening ◽  
Sickle Cell ◽  
Sickle Cell Anemia ◽  
Comprehensive Treatment ◽  
Cell Disease ◽  
Hemoglobin S ◽  
Screening Programs ◽  
Overall Mortality

Newborn screening for sickle cell disease has been recommended as a method of decreasing patient mortality. However, its effectiveness in accomplishing this has not been reliably measured. To help determine the effectiveness, 10 years of experience in newborn screening have been summarized. The effects of early patient enrollment in a comprehensive treatment program on long-term morbidity and mortality are reported. From 1975 to 1985, 84,663 newborns were screened regardless of race or ethnic background. Bart's hemoglobin was present in 5%, hemoglobin AS in 2.6%, and hemoglobin AC in 0.75%. Excluding Bart's, approximately 3.6% of all newborns were carriers for hemoglobinopathy. Sickle cell disease occurred in 1:951 births (58 hemoglobin SS, 25 hemoglobin FSC, three hemoglobin S-β+-thalassemia, and three hemoglobin S-β°-thalassemia). In addition, one in every 4,233 newborns had a clinically significant thalassemia syndrome (eight hemoglobin FE, ten hemoglobin F only, two hemoglobin H). Compared with other newborn screening programs in California, (congenital hypothyroidism, 1:3,849; phenylketonuria 1:22,474, galactosemia 1:74,103), hemoglobinopathies are the most prevalent congenital disease. Eighty-one newborns with sickle cell disease were followed for 7.2 years. Patients experienced 513 hospitalizations, including 13 episodes of sepsis with or without meningitis and ten acute sequestration crises. The overall mortality rate for patients with sickle cell anemia diagnosed in the newborn period was 1.8%. In comparison, the clinical course of 64 patients with sickle cell anemia diagnosed after 3 months of age and followed for an average of 9.4 years was analyzed. Five of these patients died. In two of these, sickle cell anemia was diagnosed at the time of the death. Overall mortality rate in this group was 8%. In summary, the data indicate that newborn screening, when coupled with extensive follow-up and education, will significantly decrease patient mortality.

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