Up to $25 million in Funding for Management of Care Transitions for Emerging Adults with Sickle Cell Disease

Thank you again for participating in the “Management of Sickle Cell Disease” workshop hosted by the Patient-Centered Outcomes Research Institute (PCORI) on March 7, 2016. Based on the results of the meeting, we would like to inform you that PCORI will be releasing a new funding announcement titled, “Management of Care Transitions for Emerging Adults with Sickle Cell Disease.” The goal of this announcement is to support patient-centered clinical effectiveness research (CER) that focuses on comparing the effectiveness of established transition coordination models for emerging adults (e.g., 16-25 years of age) with sickle cell disease transitioning from pediatric to adult care. Up to $25 million in total costs will be available to support up to three (3) studies in this area.

The mission of PCORI is to improve the quality and relevance of evidence available to help patients, caregivers, clinicians, employers, insurers, and policy makers make informed health decisions. We invite you to take a look at this announcement and share this information with others who may be interested.

The pre-announcement is available on our website

The funding announcement will be released on August 15, 2016.


Meet Alhaja Asiata Adupe – The Oldest Person Living with Sickle Cell at 90


Stem cell transplant cures children with sickle cell anemia, says Alberta hospital

To our knowledge, no one else is offering this protocol in children with sickle cell anemia,” said Guilcher, who is also an assistant professor in the departments of oncology and pediatrics at the University of Calgary’s Cumming School of Medicine.

What sets the Calgary procedure apart from other sickle cell anemia cures in young children is the lead up to the transplant.

“​This protocol uses the ‘lightest’ doses of medication — no chemotherapy but immune suppressing drugs only, with a low dose of radiation,” said Dr. Guilcher in a statement.

While the protocol was developed and is used in the U.S., Dr. Guilcher said he’s not aware of any other hospital using it on children.

More exciting is the fact that there have been no incidents of stem cell rejection


Opioids not ideal for sickle cell patients’ pain management, study says

Opioid painkillers are slowly being questioned as the best option for pain management as studies suggest they may prolong pain or make it worse, and a new study suggests the drugs may not be the best option for sickle cell disease patients who often experience excruciating pain.

Some sickle cell disease patients with chronic pain could be getting worse because of their treatment with opioids, researchers at Johns Hopkins University report in the American Journal of Preventive Medicine.


New approach could make bone marrow transplants safer  Using antibodies in place of radiation and chemotherapy

Harvard Stem Cell Institute (HSCI) scientists have taken the first steps toward developing a treatment that would make bone marrow – blood stem cell – transplantation safer and, as a result, more widely available to the millions of people living with blood disorders like sickle cell anemia, thalassemia, and AIDS.

Bone marrow transplantation currently is the only curative therapy for these blood diseases. But, for the new, transplanted stem cells to do their work, the faulty stem cells must first be “evicted” or killed. Accomplishing that requires patients endure chemotherapy and radiation — a vicious assault on the body with life-long consequences.

In a study recently published in the journal Nature Biotechnology, HSCI researchers at Harvard University and Massachusetts General Hospital (MGH), in collaboration with Boston Children’s Hospital and Dana Farber Cancer Institute, have developed a non-toxic transplantation procedure using antibodies to specifically target blood stem cells in mice, an approach they hope will make blood stem cell transplants for these patients far less toxic.  The new treatment removes more than 98% of blood stem cells, making it as effective as chemotherapy and radiation.

“Instead of using non-targeted drugs that have lots of collateral damage we thought we could take advantage of the precision of the immune system, in particular, antibodies,” said David Scadden, MD, Co-director of HSCI, the Gerald and Darlene Jordan Professor of Medicine at Harvard University, and senior author on the paper.

As part of the immune system, antibodies naturally seek and destroy foreign agents in the body. Rahul Palchaudhuri, a postdoctoral fellow in Scadden’s lab and first author on the paper, armed CD45-targeting antibodies with a payload that destroys only existing blood cells. The payload kills cells by means other than genetic destruction, in contrast to the current standard treatments.

“Antibodies are remarkably specific in what they target,” said Palchaudhuri, a chemist by training, with a background in cancer research. “We can direct them to CD45, a cell marker which is exclusively expressed in the blood system. That way we avoid toxicities to non-blood tissues.”

Unlike chemotherapy and radiation — which indiscriminately damage cells and tissues, healthy or otherwise — the CD45-targeting antibodies leave the thymus and the bone marrow, environments critical to the formation of T cells and innate immune cells, unharmed. Animals receiving the antibody treatment were able to withstand infection that was lethal to mice treated with radiation. Currently, infections after transplant are common and may be severe, causing death in a substantial number of people.

About one in ten patients do not survive transplantation following the standard treatments. Those who do may suffer from stunted growth and intellectual development, infertility, and damaged DNA; at present, patients can only attempt a curative transplant by increasing their risk of developing cancer later.

Because of this, families and doctors often shrink from transplant options, particularly when it comes to treating children, and it will limit the extent to which the breakthroughs in gene therapy and gene editing will be applied, explained Scadden, who is a practicing hematologist at MGH and chairman of Harvard’s Department of Stem Cell and Regenerative Biology.

Animals that received the antibody treatment had a broad ten-day window within which they could accept a bone marrow transplant, and individuals that did not receive a bone marrow transplant were able to fully recover without adverse effects. Furthermore, mice suffering from sickle cell anemia were successfully transplanted using the antibody method and cured of their anemia. Should the same hold true for humans, what amounts to months of recovery in a hospital bed may be replaced by an outpatient procedure, and a failed transplant would not be fatal.

“If this approach works in humans, it will really change the conversation that providers have with patients,” Scadden said, especially for those “who have these underlying genetic disorders and for who the new gene-editing and gene therapy techniques are being developed.”  The scientists are now trying to identify antibodies that would be effective in humans, and a company has been formed to move the work towards translation and determine which models are most useful in a preclinical setting.

“It brings precision medicine into the area of transplant in a way that hasn’t been there and is needed,” Scadden said.


New Video Resource – This is “The Year of Clinical Trial Diversity”!

As you know, we have been working to develop some new resources to help promote diversity in clinical trials. We now have five new public service announcements, a blog, and an infographic that you can use to encourage minorities to join clinical trials. The videos feature Shirley Miller, who is living with sickle cell disease, or Dr. Luciana Borio, Acting Chief Scientist at FDA. They share their views on why diversity in clinical trials matter and encourage patients to learn more. This is important because sometimes minorities may respond differently to medical products. If participation remains low, disparities may continue to exist because of lack of available information about differences in response to treatments- particularly for diseases where minorities are adversely impacted.

We have developed a communications toolkit to help you become a “Clinical Trials Champion” in sharing this important message with your community. In the attached toolkit, you will find draft social media messages, sample email/newsletter and blog, and graphics to help promote these efforts.

Let’s inspire minority audiences to learn more about clinical trials! Commissioner Rob Califf, M.D. dedicated 2016 to improving minority participation in clinical trials. I hope that you can join us in promoting this effort. For more information about this initiative, please contact me at

The videos and resources are available here:

Video 1: Shirley’s Story: How to Find Information about Clinical Trials


Video 2: Shirley’s Story: Getting Access to Cutting Edge Therapies


Video 3: Shirley’s Story: You Don’t Have to be Sick to Participate


Video 4: Shirley’s Story: Diversity is Critical to Making Better Medical Products


Video 5: Shirley’s Story: Diversity is Critical to Making Better Medical Products (LONG)


Video 6: Dr. Luciana Borio: FDA’s Role in Increasing Clinical Trial Diversity


Infographic: 4 Ways to Be a #ClinicalTrialsChampion


Dr. Jonca Bull’s Blog: Be A Champion for Clinical Trial Diversity


Interview with Cariny Nunez, FDA Public Health Advisor [Spanish]

Link: (part 1) Link: (part 2)


The CDC Celebrates World Sickle Cell Day with Launch of New Resources!

Sickle Cell Disease is featured as the Disease of the Week in the CDC’s tablet application. The application is available for download on iOS and android operating systems. The content is also available on the CDC website. There, users can learn key facts, prevention tips, and take a quick quiz to test their knowledge about Sickle Cell Disease.

In addition the CDC has added to their Sickle Cell Trait Toolkit which includes a collection of resources to increase understanding of Sickle Cell Trait


Sickle Cell Disease Isn’t Laughable, But It Has a Comedic Ambassador — Kier “Junior” Spates

Kier “Junior” Spates is known for his ability to make audiences laugh on his comedy tour and his regular appearances on “The Steve Harvey Morning Show.” However, on “Beyond Just Talk with S. L. Young,” Mr. Spates shared his private battle with sickle cell disease that isn’t laughable. As he says in his signature tagline “This Just Got Serious.”


Sickle cell in the Medical Literature


Clin J Pain. 2016 Jun 17. [Epub ahead of print]

Widespread Pain among Youth with Sickle Cell Disease Hospitalized with Vasoocclusive Pain: A Different Clinical Phenotype?

Zempsky WT1, Wakefield EO, Santanelli JP, New T, Smith-Whitley K, Casella JF, Palermo TM.



The purpose of this study was to describe the clinical phenotype of widespread pain among youth with sickle cell disease (SCD) hospitalized with vasoocclusive pain.


156 youth with SCD, between 7-21 years of age hospitalized at 4 children’s hospitals for a vasoocclusive episode (VOE) were evaluated. Data were collected during one day of the hospitalization.


Using the 2010 American College of Rheumatology guidelines, 21.8% of patients were identified as having widespread pain (pain in 7 or more unique body locations). Patients classified as having widespread pain had higher pain intensity (6.5 vs. 5.6; t=2.19, P=0.03) higher pain burden (13.0 vs. 9.8; t=3.09, P=0.002), higher acute functional disability (22.1 vs. 16.5; t=2.43, P=0.016), higher chronic functional disability (30.4 vs. 22.2; t=2.31,P=0.02), lower positive affect (22.9 vs. 27.6; t=2.23, P=0.027), and lower quality of life (56.2 vs. 62.9; t=1.99,P=0.049) than those youth with SCD without widespread pain.


Assessment of widespread pain may identify a unique clinical phenotype of youth with SCD with differing treatment needs.

PMID: 27322398 [PubMed – as supplied by publisher]



Am J Prev Med. 2016 Jul;51(1 Suppl 1):S87-98. doi: 10.1016/j.amepre.2016.01.016.

Community Health Workers as Support for Sickle Cell Care.

Hsu LL1, Green NS2, Donnell Ivy E3, Neunert CE4, Smaldone A4, Johnson S5, Castillo S1, Castillo A1, Thompson T6, Hampton K7, Strouse JJ8, Stewart R8, Hughes T9, Banks S10, Smith-Whitley K11, King A12, Brown M13, Ohene-Frempong K11, Smith WR5, Martin M1.


Community health workers are increasingly recognized as useful for improving health care and health outcomes for a variety of chronic conditions. Community health workers can provide social support, navigation of health systems and resources, and lay counseling. Social and cultural alignment of community health workers with the population they serve is an important aspect of community health worker intervention. Although community health worker interventions have been shown to improve patient-centered outcomes in underserved communities, these interventions have not been evaluated with sickle cell disease. Evidence from other disease areas suggests that community health worker intervention also would be effective for these patients. Sickle cell disease is complex, with a range of barriers to multifaceted care needs at the individual, family/friend, clinical organization, and community levels. Care delivery is complicated by disparities in health care: access, delivery, services, and cultural mismatches between providers and families. Current practices inadequately address or provide incomplete control of symptoms, especially pain, resulting in decreased quality of life and high medical expense. The authors propose that care and care outcomes for people with sickle cell disease could be improved through community health worker case management, social support, and health system navigation. This paper outlines implementation strategies in current use to test community health workers for sickle cell disease management in a variety of settings. National medical and advocacy efforts to develop the community health workforce for sickle cell disease management may enhance the progress and development of “best practices” for this area of community-based care.

Copyright © 2016 American Journal of Preventive Medicine. All rights reserved.

PMCID: PMC4918511 [Available on 2017-07-01] Free Article

PMID: 27320471 [PubMed – in process]




Am J Prev Med. 2016 Jul;51(1 Suppl 1):S78-86. doi: 10.1016/j.amepre.2016.01.024.

Mistrust of Pediatric Sickle Cell Disease Clinical Trials Research.

Stevens EM1, Patterson CA2, Li YB2, Smith-Whitley K2, Barakat LP2.



Sickle cell disease (SCD) research is hampered by disparities in participation due in part to mistrust of research among racial/ethnic minorities. Beyond the historic context of research misconduct, little is known about the associations of social ecologic factors with mistrust and of mistrust with SCD clinical trials enrollment. This study evaluated proximal (age, gender, disease severity, perceived stress, SES) and distal (religious beliefs, social support, instrumental support) factors related to mistrust of research among caregivers of children with SCD and adolescents and young adults (AYAs) with SCD.


Over an 18-month period (2009-2010), participants completed questionnaires of perceived barriers and benefits to clinical trials enrollment, perceived stress, and self-reported demographic and disease-related information. Analyses (January-June 2015) used multivariable linear regressions to evaluate predictors of mistrust.


Data were analyzed for 154 caregivers (mean age, 38.75 years; SD=9.56 years; 90.30% female) and 88 AYAs (mean age, 24.76 years; SD=7.25 years; 46.40% female). Among caregivers (full model, R(2)=0.14, p≤0.001), greater mistrust was explained by higher perceived stress (β=0.04, p=0.052); religious beliefs (β=0.61, p≤0.001); and greater instrumental support (β=0.07, p=0.044). Among AYAs (full model, R(2)=0.18, p≤0.001), higher mistrust was explained by being male (β=-0.56, p≤0.001) and lower instrumental support (β=-0.11, p=0.016). Mistrust was significantly greater among caregivers that reported no prior involvement in medical research (p=0.003).


By understanding the complexity through which social ecologic factors contribute to mistrust, researchers may create targeted strategies to address mistrust and increase engagement in SCD research for caregivers and AYAs.

Published by Elsevier Inc.

PMCID: PMC4916926 [Available on 2017-07-01] Free Article

PMID: 27320470 [PubMed – in process]



Am J Prev Med. 2016 Jul;51(1 Suppl 1):S69-77. doi: 10.1016/j.amepre.2016.02.012.

Chronic Opioid Therapy and Central Sensitization in Sickle Cell Disease.

Carroll CP1, Lanzkron S2, Haywood C Jr2, Kiley K3, Pejsa M3, Moscou-Jackson G4, Haythornthwaite JA3, Campbell CM3.


Chronic opioid therapy (COT) for chronic non-cancer pain is frequently debated, and its effectiveness is unproven in sickle cell disease (SCD). The authors conducted a descriptive study among 83 adult SCD patients and compared the severity of disease and pain symptoms among those who were prescribed COT (n=29) with those who were not using COT. All patients completed baseline laboratory pain assessment and questionnaires between January 2010 and June 2014. Thereafter, participants recorded daily pain, crises, function, and healthcare utilization for 90 days using electronic diaries. Analyses were conducted shortly after the final diary data collection period. Patients on COT did not differ on age, sex, or measures of disease severity. However, patients on COT exhibited greater levels of clinical pain (particularly non-crisis); central sensitization; and depression and increased diary measures of pain severity, function, and healthcare utilization on crisis and non-crisis diary days, as well as a greater proportion of days in crisis. Including depressive symptoms in multivariate models did not change the associations between COT and pain, interference, central sensitization, or utilization. Additionally, participants not on COT displayed the expected positive relationship between central sensitization and clinical pain, whereas those on COT demonstrated no such relationship, despite having both higher central sensitization and higher clinical pain. Overall, the results point out a high symptom burden in SCD patients on COT, including those on high-dose COT, and suggest that nociceptive processing in SCD patients on COT differs from those who are not.

Copyright © 2016 American Journal of Preventive Medicine. Published by Elsevier Inc. All rights reserved.

Free Article

PMID: 27320469 [PubMed – in process]




Am J Prev Med. 2016 Jul;51(1 Suppl 1):S62-8. doi: 10.1016/j.amepre.2016.03.003.

Improved Guideline Adherence With Integrated Sickle Cell Disease and Asthma Care.

McClain BL1, Ivy ZK2, Bryant V3, Rodeghier M4, DeBaun MR3.

Author information:



In children with sickle cell disease (SCD), concomitant asthma is associated with increased morbidity and mortality when compared with children with SCD without asthma. Despite the well-established burden of asthma in children with SCD, no paradigm of care exists for the co-management of these two diseases.


To address this gap, an integrated SCD and asthma clinic was created in a community health center that included (1) a dual respiratory therapist/asthma case manager; (2) an SCD nurse practitioner with asthma educator certification; (3) an onsite pulmonary function test laboratory; (4) a pediatric hematologist with expertise in managing SCD and asthma; and (5) application of the National Asthma Education and Prevention Program guidelines. A before (2010-2012) and after (2013-2014) study design was used to assess for improved quality of care with implementation of an integrative care model among 61 children with SCD and asthma followed from 2010 to 2014.


Asthma action plan utilization after initial diagnosis increased with the integrative care model (n=16, 56% before, 100% after, p=0.003), as did the use of spirometry in children aged ≥5 years (n=41, 65% before, 95% after, p<0.001) and correction of lower airway obstruction (n=10, 30% before, 80% after, p=0.03).


Although the use of an integrative care model for SCD and asthma improved evidence-based asthma care, longer follow-up and evaluation will be needed to determine the impact on SCD-related morbidity.

Copyright © 2016 American Journal of Preventive Medicine. Published by Elsevier Inc. All rights reserved.

Free Article

PMID: 27320468 [PubMed – in process]


Am J Prev Med. 2016 Jul;51(1 Suppl 1):S55-61. doi: 10.1016/j.amepre.2016.01.023.

Care Coordination for Children With Sickle Cell Disease: A Longitudinal Study of Parent Perspectives and Acute Care Utilization.

Rattler TL1, Walder AM2, Feng H2, Raphael JL3.



Care coordination (CC), a core element of the medical home, has the potential to reduce fragmented care and improve patient experience for children with sickle cell disease (SCD). This study aimed to (1) assess CC for pediatric SCD and (2) determine its association with acute care utilization-emergency department encounters and hospitalizations. It was hypothesized that CC would reduce acute care utilization.


A longitudinal study of 101 children with SCD was conducted. Parents completed a survey instrument on enrollment. Utilization chart review was conducted 9 months post survey. Outcome variables were emergency department encounters and hospitalizations. Independent variables were parent-reported CC, satisfaction with communication between healthcare providers, and satisfaction with communication between healthcare providers and non-medical providers (e.g., schools, child care centers). Multivariate negative binomial regression was conducted to assess associations between CC and acute care utilization. Data were collected in 2011-2013 and analyzed in 2015.


One third of children had emergency department encounters and 30% had hospitalizations. At enrollment, 25% of parents reported receiving CC help and 20% reported need for extra CC. Most parents were satisfied with communication between physicians but only two thirds were satisfied with communication between their healthcare providers and non-medical providers. No significant associations were found between CC measures and acute care utilization.


Although parents report multiple CC deficiencies, no associations were found between CC and acute care utilization. Population-based studies are warranted to more definitively determine the association between CC and acute care utilization for children with SCD.

Copyright © 2016 American Journal of Preventive Medicine. Published by Elsevier Inc. All rights reserved.

PMCID: PMC4916339 [Available on 2017-07-01] Free Article

PMID: 27320467 [PubMed – in process]




Am J Prev Med. 2016 Jul;51(1 Suppl 1):S5-9. doi: 10.1016/j.amepre.2016.03.008.

Sickle Cell Disease, 2015: A Patient Advocate’s Perspective.

Williams AM1, Smith-Whitley K2.

Author information:

Free Article

PMID: 27320466 [PubMed – in process]




Am J Prev Med. 2016 Jul;51(1 Suppl 1):S31-8. doi: 10.1016/j.amepre.2016.01.001.

Hydroxyurea Use in Young Children With Sickle Cell Anemia in New York State.

Anders DG1, Tang F2, Ledneva T3, Caggana M4, Green NS5, Wang Y6, Sturman LS7.



This study examined hydroxyurea usage in young children with sickle cell anemia within New York State (NYS). The cohort was 273 children with sickle cell anemia born in NYS in 2006-2009 and enrolled essentially continuously in Medicaid for the first 4 years of life.


Medicaid data were used to examine hydroxyurea usage in this group by age at first prescription fill, persistence, region, treatment institution, and year. Log-binomial regression models were used to estimate the likelihood of receiving hydroxyurea treatment. Data from birth through 2014 for all members of the study group were assembled and analyzed in 2015.


About 25% of the cohort had at least one filled hydroxyurea prescription by their fifth birthday, and nearly 40% by the end of the study period. The mean proportion of days covered for the first year of therapy was 56.3%. Adherence was also assessed by calculating medication possession ratios for individual treatment periods. Slightly more than one third of treated children showed 80% coverage by these measures. There was a consistent, but not statistically significant, trend toward younger age at first fill. Significant regional and treatment center differences in initiation of hydroxyurea use, but not in persistence after initiation, were noted among NYS centers.


Subsequent to clinical studies demonstrating safety, current NYS-wide use of hydroxyurea in young children with sickle cell anemia appears to be widespread and increasing. However, practice differences between treatment centers and inadequate adherence may limit the full disease-modifying effects of hydroxyurea.

Copyright © 2016 American Journal of Preventive Medicine. Published by Elsevier Inc. All rights reserved.

Free Article

PMID: 27320463 [PubMed – in process]




Am J Prev Med. 2016 Jul;51(1 Suppl 1):S39-47. doi: 10.1016/j.amepre.2016.02.019.

Newborn Screening Programs and Sickle Cell Disease: A Public Health Services and Systems Approach.

Minkovitz CS1, Grason H2, Ruderman M2, Casella JF3.



Despite universal newborn screening (NBS), children in the U.S. continue to experience morbidity and mortality from sickle cell disease and related causes. Recognizing that assessments of public health services and systems can improve public health system performance and ultimately health outcomes, this study examined variations in NBS program activities for sickle cell disease.


A mixed methods study included (1) a 2009 survey of NBS programs based on ten essential public health services (N=39 states with ten or more sickle cell births over a 3-year period) and (2) key informant interviews in 2011 with 13 states that had sufficient Phase 1 survey scores, black births, and variability in state legislation and geography. Key informants were from 13 NBS programs, 22 sickle cell treatment centers, and ten advocacy organizations. Analyses were conducted in 2009-2014.


Considerable variability exists across states in program activities and roles. More programs reported activities oriented to care of individuals-ensuring access to services, coordination, and provider education; fewer reported planning and analysis activities oriented to statewide policy development and system change. Numbers of activities were not related to the number of affected births. In-depth interviews identified opportunities to enhance activities that support statewide comprehensive systems of care.


NBS programs perform important public health roles that complement and enhance clinical services. Nationwide efforts are needed to enable NBS programs to strengthen population-based functions that are essential to ensuring quality of care for the entire population of children and families affected by sickle cell disease.

Copyright © 2016 American Journal of Preventive Medicine. Published by Elsevier Inc. All rights reserved.

PMCID: PMC4916337 [Available on 2017-07-01] Free Article

PMID: 27320464 [PubMed – in process]




Am J Prev Med. 2016 Jul;51(1 Suppl 1):S3-4. doi: 10.1016/j.amepre.2015.10.018.

Sickle Cell Disease: A Roadmap for Getting to Excellence Everywhere.

Homer CJ1, Oyeku SO2.

Free Article

PMID: 27320462 [PubMed – in process]




Am J Prev Med. 2016 Jul;51(1 Suppl 1):S24-30. doi: 10.1016/j.amepre.2016.02.011.

Determining Adherence to Quality Indicators in Sickle Cell Anemia Using Multiple Data Sources.

Neunert CE1, Gibson RW2, Lane PA3, Verma-Bhatnagar P4, Barry V3, Zhou M5, Snyder A6.



Advances in primary prophylaxis have resulted in improved outcomes for patients with sickle cell anemia (SCA; i.e., hemoglobin SS- and Sβ(0)-thalassemia). Standard prophylactic measures include a first pneumococcal polysaccharide vaccine (PPV) and transcranial Doppler ultrasound (TCD) at age 2 years. Though efficacious, evidence suggests that delivery of these interventions is suboptimal. This study reports adherence to these measures and examines concordance across various data sources, using Registry and Surveillance for Hemoglobinopathies project data.


Retrospective database and SCA center chart review identified children with SCA aged 24-36 months between January 1, 2004, and December 31, 2008. PPV and TCD administration were determined through Medicaid and Children’s Health Insurance Program administrative claims data, medical record review, and Georgia Registry of Immunization Transaction and Services. Analysis was conducted in 2015.


A total of 125 children met inclusion criteria. Forty-five (36.0%) children had documentation of both interventions, whereas 19 (15.2%) had no documentation of either intervention. Sixty-one (48.8%) children obtained only one intervention. Of these, more were likely to have had PPV than TCD (77.0% vs 23.0%, respectively, p<0.001). Agreement between claims data and medical record review was moderate for PPV (κ=0.55) and substantial for TCD (κ=0.74).


No single, reliable data source for tracking standard of care for children with SCA statewide was found. According to study data, prophylaxis measures were not universally implemented during the surveillance period. Further research is needed to adequately track changes over time, determine risk groups, and develop methods of evaluating important metrics.

Copyright © 2016 American Journal of Preventive Medicine. Published by Elsevier Inc. All rights reserved.

PMCID: PMC4918094 [Available on 2017-07-01] Free Article


PMID: 27320461 [PubMed – in process]


Similar articles



Am J Prev Med. 2016 Jul;51(1 Suppl 1):S17-23. doi: 10.1016/j.amepre.2016.02.004.

Improving Sickle Cell Transitions of Care Through Health Information Technology.

Frost JR1, Cherry RK2, Oyeku SO3, Faro EZ4, Crosby LE5, Britto M5, Tuchman LK6, Horn IB7, Homer CJ8, Jain A2.



Transitions between inpatient and outpatient care and pediatric to adult care are associated with increased mortality for sickle cell disease (SCD) patients. As accurate and timely sharing of health information is essential during transitions, a health information technology (HIT)-enabled tool holds promise to improve care transitions.


From 2012 through 2014, the team conducted and analyzed data from an environmental scan, key informant interviews, and focus groups to inform the development of an HIT-enabled tool for SCD patients’ use during care transitions. The scan included searches of peer-reviewed and gray literature to understand SCD patient needs, transition concerns, and best practices in mobile health applications, and searches of websites and online stores to identify existing transition tools and their features. Eleven focus groups consisted of four groups of SCD patients of varying ages (≥9 years); three groups of parents/caregivers of SCD patients; three groups of providers; and one with IT developers.


In focus groups, patients and caregivers reported that the transition from home to the emergency department (ED) was the most challenging; the ED was also where transitions from pediatric to adult care usually occurred. Patients felt they were not taken seriously by unfamiliar ED providers, and their inability to convey their diagnosis, pain regimen, and detailed medical history while in significant pain hindered care.


The environmental scan did not reveal an existing suitable transition tool, but patients, parents, providers, and IT experts saw the potential and appeal of creating a tool to meet ED health information needs to improve care transitions.

Copyright © 2016 American Journal of Preventive Medicine. All rights reserved.

Free Article


PMID: 27320460 [PubMed – in process]




Am J Prev Med. 2016 Jul;51(1 Suppl 1):S10-6. doi: 10.1016/j.amepre.2016.01.021.

Implementation of a Process for Initial Transcranial Doppler Ultrasonography in Children With Sickle Cell Anemia.

Crosby LE1, Joffe NE2, Davis B3, Quinn CT2, Shook L2, Morgan D3, Simmons K3, Kalinyak KA2.


Stroke, a devastating complication of sickle cell anemia (SCA), can cause irreversible brain injury with physical and cognitive deficits. Transcranial Doppler ultrasonography (TCD) is a non-invasive tool for identifying children with SCA at highest risk of stroke. National guidelines recommend that TCD screening begin at age 2 years, yet there is research to suggest less than half of young children undergo screening. The purpose of this project was to use quality improvement methods to improve the proportion of patients aged 24-27 months who successfully completed their initial TCD from 25% to 75% by December 31, 2013. Quality improvement methods (e.g., process mapping, simplified failure mode effect analysis, and plan-do-study-act cycles) were used to develop and test processes for identifying eligible patients, scheduling TCDs, preparing children and families for the first TCD, and monitoring outcomes (i.e., TCD protocol). Progress was tracked using a report of eligible patients and a chart showing the age in months for the first successful TCD (population metric). As of December 2013, 100% of eligible patients successfully completed their initial TCD screen; this improvement was maintained for the next 20 months. In November 2014, a Welch’s one-way ANOVA was conducted. Results showed a statistically significant difference between the average age of first TCD for eligible patients born in 2009 and eligible patients born during the intervention period (2010-2013; F[1,11.712]=16.03, p=0.002). Use of quality improvement methods to implement a TCD protocol was associated with improved TCD screening rates in young children with SCA.

Copyright © 2016 American Journal of Preventive Medicine. Published by Elsevier Inc. All rights reserved.

Free Article

PMID: 27320459 [PubMed – in process]




Am J Prev Med. 2016 Jul;51(1 Suppl 1):S1-2. doi: 10.1016/j.amepre.2015.11.002.

Sickle Cell Disease: A Continued Call to Action.

Hassell KL1.

Author information:

Free Article

PMID: 27320458 [PubMed – in process]




Hum Genet. 2016 Jun 17. [Epub ahead of print]

Treating hemoglobinopathies using gene-correction approaches: promises and challenges.

Cottle RN1, Lee CM2, Bao G3.


Hemoglobinopathies are genetic disorders caused by aberrant hemoglobin expression or structure changes, resulting in severe mortality and health disparities worldwide. Sickle cell disease (SCD) and β-thalassemia, the most common forms of hemoglobinopathies, are typically treated using transfusions and pharmacological agents. Allogeneic hematopoietic stem cell transplantation is the only curative therapy, but has limited clinical applicability. Although gene therapy approaches have been proposed based on the insertion and forced expression of wild-type or anti-sickling β-globin variants, safety concerns may impede their clinical application. A novel curative approach is nuclease-based gene correction, which involves the application of precision genome-editing tools to correct the disease-causing mutation. This review describes the development and potential application of gene therapy and precision genome-editing approaches for treating SCD and β-thalassemia. The opportunities and challenges in advancing a curative therapy for hemoglobinopathies are also discussed.

PMID: 27314256 [PubMed – as supplied by publisher]



Sickle Cell Conferences and Events


6th International African Symposium on Sickle Cell Disease

July 11th – 15th, 2016

Labadi Beach Hotel, Accra, Ghana

The Symposium website: is now live.  Please check it often for announcements and updates.

Please visit our website: to download the Registration form


Sickle Cell 101 is hosting at the 2016 Sickle Cell Disease Patient and Family Convention July 28 – 31 in Hollywood California

Venue: Loews Hollywood Hotel; 1755 Highland Ave, Los Angeles, CA 90028

For more information about the Sickle Cell Disease Patient and Family Convention, please visit:


Sickle Cell Education Day 2016 –Children’s Healthcare of Atlanta, GA

“Learning to Live, Love, and Hope with Sickle Cell Disease” 

Date: Saturday, September 24, 2016

Time: 9:00 a.m. to 3:00 p.m.


Parking: Courtyard by Marriott, Downtown Decatur
130 Clairemont Avenue
Decatur, GA 30030

Onsite parking is available for $8.

Complimentary parking will be available at the below address:
First Baptist Church of Decatur
308 Clairemont Ave.
Decatur, GA 30030

Registration for this program opens June 22, 2016. Please contact for additional information.


Sickle Cell Partners of the Carolinas presents, Sickle Cell Disease, “Let’s talk about it” day conference

Saturday September 10, 2016

9 am to 2 pm

Friendship Missionary Baptist Church Conference Center

3400 Beatties Ford Road, Charlotte NC 28216


Calling All Abstracts!

Submit your abstracts for the 44th SCDAA annual convention Baltimore, MD on September 27-October 1st, 2016

Interested in highlighting your work as a community based member organization, physician, nurse, social worker, or other team working on behalf of people with sickle cell disease and their families? Submit an abstract on the work that you or your team has completed or is in the process of completing for an opportunity to share at the 44th Annual Sickle Cell Convention. This year’s convention will be held in the great city of Baltimore, MD on September 27-October 1st, 2016 at the Hyatt Regency hotel. For more information on abstracts or to submit your application clicking HERE.

SCDAA 44th Annual Convention Sept 27 – Oct 1 Baltimore MD



Listserv  address to join or leave: