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Stopping the Sickle Cycle – Where are we?

By Allan Platt PA-C, MMSc

In 1977, the published life expectancy for a sickle cell disease patient was mid teens. Forty years later, in 2017, life expectancy in the US has grown with identification of patients with newborn screening programs, prophylactic penicillin from birth to age six, the advent of the pneumococcal vaccine, transcranial doppler ultrasound screening with transfusions to prevent stroke, chest syndrome recognition and prevention, comprehensive pediatric care and hydroxyurea. In 1982, a patient at St. Jude Children’s Research Hospital in Memphis TN had leukemia and sickle cell disease. A St. Jude doctor performed a bone marrow transplant using marrow donated from the patient’s brother.  The patient was cured of both leukemia and sickle cell disease. Many stem cell transplants have been curative with a 90% survival but only 18% of patients eligible have an acceptable match. The multicenter trials of  hydroxyurea in the 1980s and 90s as preventive therapy reduced the need for blood transfusions, pain events and hospitalizations. It was found to prolong lives. This was approved for medical use by the FDA in 1998. By the early 1990s, the Cooperative Study of Sickle Cell Disease estimated a median life expectancy of those with sickle cell anemia, the most severe form of the disease, of 42 years of age for males and 48 years of age for females. In 2005 Lanzkron et al confirmed these findings of a significant decrease in mortality for children with SCD, but mortality for adults increased during the same time period. This may reflect a lack of access to high-quality comprehensive care for adults with SCD in the US.

In 2017 two notable advances happened: In July the FDA approved Endari or L-glutamine oral powder to Emmaus Medical Inc for use as a preventative in sickle cell patients 5 years and older. The safety and efficacy of Endari were studied in a randomized trial of patients ages five to 58 years old with sickle cell disease who had two or more painful crises within the 12 months prior to enrollment in the trial. Patients were assigned randomly to treatment with Endari or placebo, and the effect of treatment was evaluated over 48 weeks. Patients who were treated with Endari experienced fewer hospital visits for pain treated with a parenterally administered narcotic or ketorolac, on average, compared to patients who received a placebo, fewer hospitalizations for sickle cell pain (median 2 vs. median 3), and fewer days in the hospital (median 6.5 days vs. median 11 days).  Patients who received Endari also had fewer occurrences of acute chest syndrome compared with patients who received a placebo (8.6 percent vs. 23.1 percent).

The March 2nd issue of the New England Journal of Medicine published Gene Therapy in a Patient with Sickle Cell Disease, a report of the first successful case of using gene therapy in a patient with sickle cell disease in Paris, France. Gene therapy that delivered an antisickling variant of hemoglobin in an autologous hematopoietic stem cell (HSC) transplant had ameliorated all the symptoms of severe sickle cell disease (SCD) in a 15-year-old boy 2 years out from the procedure.

These two major events bring hope to all those battling the sickle cycle of recurrent pain and complications. Currently, there are 198 active research studies listed at We have come a long way in 40 years but more is needed to bring a cure for all.


Platt OS, Brambilla DJ, Rosse WF, Milner PF, Castro O, Steinberg MH, et al. Mortality in sickle cell disease. Life expectancy and risk factors for early death. N Engl J Med. 1994;330:1639–44.

Lanzkron S, Carroll CP, Haywood C. Mortality Rates and Age at Death from Sickle Cell Disease: U.S., 1979–2005. Public Health Reports. 2013;128(2):110-116.


Making Advances Against Sickle Cell Disease

By: Patricia Oneal, M.D., and Rosanna Setse, M.D., M.P.H., Ph.D.

L-glutamine oral powder

N Engl J Med 2017; 376:848-855 March 2, 2017



Sickle Cell Disease Update form the ASH meeting in December 27, 2017



Sickle cell patients fight uphill battle for research, treatment — and compassion

By Jenny Gold — Kaiser Health News  December 26, 2017



Sickle Cell Disease Advocacy In Ghana: Experiences From An African Country

Gifty Tina Naa Ayeley Mensah1, Aba Bentil Andam, Charlotte Owusu, Kwaku Kwateng Yeboah4, Loretta Kwateng Yeboah4.

  1. Ministry of Health, Republic of Ghana, Accra , Ghana – West Africa
  1. Ghana Academy of Arts and Sciences –
  1. Sickle Cell Condition Advocates (SICCA), Ghana.

Corresponding Author: Email: Website www.sicklecare.wixsite,com/sicca

Sickle Cell Disease (SCD) is the most common serious genetic disease in Africa. Ghana and other countries in West and Central Africa have the highest prevalence of SCD and related disorders in the world. In Ghana about 2% of newborns have sickle cell disease which translates to about 16,000 to 18,000 children born annually with SCD, with 20% – 25 % born as trait or carriers.

Barriers to Quality Health Care Services in Ghana include:

  1. Lack of SCD Clinics in hospitals to provide specialist care for sickle cell patients.
  2. Lack of capacity building of Health Care Workers eg. doctors, nurses, laboratory technologists, genetic counselors and health educators to effectively manage SCD patients.
  3. Need for development of treatment protocols, guidelines and training modules for holistic management of the disease.
  4. Lack of laboratory services to provide highly sensitive and specific diagnostic facilities at all regional hospitals and a basic package for district hospitals to facilitate prompt diagnosis and quality management.


Ghana as one of the countries with the highest prevalence of SCD and other related disorders in the world calls for a holistic attempt in providing a clinical, health educational, social and psychological care for affected as well as unaffected population for all to make informed choices.

The Sickle Cell Condition Advocates (SICCA), a registered Non-Governmental Organization (NGO) which has been in operation since September 2007 is involved in many activities which aims at educating, counseling, raising awareness on SCD and reducing it to the barest minimum in the long term. It looks at the challenges across all aspects of the sickle cell condition; from pre-conception to living and caring for people with the sickle cell condition and their families.

This paper is a report on Sickle Cell Advocacy Project in the three Northern regions of Ghana,     executed by by SICCA in collaboration with the Ministries of Health and Education and endorsed by the Ghana Academy of Arts and Sciences.

The objectives were as follows:

  1. To collaborate with all stakeholders to create awareness of the SCD since it affects the whole community;
  2. To initiate pre-conception screening project for people in Ghana and other developing countries.
  3. To assist in the establishment of sickle cell clinics and support groups in the various hospitals and health centers.


Formal Education: The approach used was institution based formal health education. Training sessions were organized for coordinators and volunteers in each region. Education on SCD was given to students in almost all Health Training Institutions and Senior High Schools in the three regions.

Informal Education: The approach used was community based health education. Mass media education and public events were also done, supported by the Ministries of Health and Education using publicity drive: Radio discussion programs, Talk Shows, Community/Town Hall Meetings and Information van community announcements. TV and Billboard adverts, Banners, Flyers, Posters, Presentations using Projector and Screen and ‘T’- shirts were all used to intensify the advocacy.

The promotion involved the Regional Coordinating Councils, Ministry of Information, Department of Social Welfare, Media Houses and some Community Leaders in all the three regions. Students were used for Blood Donation and Eye Screening exercises to create awareness  as well.


Eighty-six (86) health professionals from the three regions were trained in Tamale, by six resource persons from the biggest hospital in Ghana, Korle-Bu Teaching Hospital and the General Hospital in Tema.

Two coordinators were trained in each region, one from Ministry of Health and the other from Ministry of Education. Faith groups, Social groups, Youth Clubs and Fun Associations in the Tamale, Bolgatanga and Wa Municipalities, were given the advocacy message.

A room was rehabilitated for the Tamale Sickle Cell Clinic. In Bolgatanga and Wa, all rooms were ready and furnished by the Regional Hospitals. Counseling Units were set up and attached to Sickle Cell Clinics in the three Regional Hospitals. The Counseling Units started running before official opening of clinics which was done at the end of the project. All official opening ceremonies were covered by press. This was a way of creating awareness of SCD.

Advocacy groups made up of patients, parents and people with interest in SCD were established in all three northern regions.

We found in our survey the following; lack of knowledge, compounded by myths and superstitions in most localities but with knowledge on SCD, this got rid of all such concepts.


There were lack of vehicles for the project. This was solved by the use of vehicles from the Regional Hospital, Regional Health Directorate and Ministry of Education in regions. Public transport was also used some times. Another challenge that came up was the lack of staff for the project, since some of the trained personnel for the project had to discontinue work in the north. This was also solved by using volunteers and coordinators from the Regional Hospitals and Regional Health Directorate who were given some form of training in each region before work started.


Great changes in Sickle Cell awareness was created in three northern regions of Ghana through this Advocacy Project. With the availability of funds and similar efforts in other regions and adoption of recommendations, there is genuine prospect of greater countrywide change on the horizon. People saw the need for screening to know their sickle cell status to make informed choices before having children. This is the only way out for developing countries.


  1. Structured training programs for health professionals on latest Protocols in the management of  SCD.
  1. Organize “Know Your Sickle Cell Status” campaign at all educational levels right from Basic Schools.
  2. Provide/Solicit logistics and financial support or supplement for such programs in future in remaining regions in the Ghana.-
  3. SICCA is advocating that this project be executed in countries where SCD is a challenge.



New Resource – Blood Transfusion in Sickle Cell Educational videos

The Centers for Disease Control and Prevention (CDC) along with the Georgia Health Policy Center (GHPC) are pleased to announce a four-part educational video series for healthcare providers. The series, which will be housed on GHPC’s website, is designed to provide information about the strategies for reducing transfusion complications in people with sickle cell disease (SCD). Weblink

Video topics:

  1. Introduction by James Eckman, M.D.
  2. Module 1. Use of blood transfusion during acute illness by Peter Lane, M.D. 
  3. Module 2. Delayed hemolytic transfusion reactions by Ross Fasano, M.D.
  4. Module 3. Management of chronic transfusion by James Eckman, M.D.



ASH Sickle Cell Disease Initiative Website



Articles in the Medical Literature


Am J Hematol. 2017 Dec 20. doi: 10.1002/ajh.25013. [Epub ahead of print]

Sleep disordered breathing does not predict acute severe pain episodes in children with sickle cell anemia.

Willen SM1, Rodeghier M2, Rosen CL3, DeBaun MR1.


Conflicting evidence has suggested that low mean nocturnal hemoglobin oxygen saturation (SpO2 ) predicts future hospital days for acute severe pain in children with sickle cell anemia (SCA). In an unselected multi-center prospective cohort study, we tested the hypothesis that either low mean nocturnal SpO2 or high obstructive apnea-hypopnea index (OAHI; the number of obstructive apneas and hypopneas with ≥ 3% desaturation or arousal per hour of sleep) or high oxygen desaturation index (ODI; number of ≥ 3% desaturation from baseline saturation per hour of sleep) is associated with increased incidence rates of pain. A total of 140 children with SCA with a median age of 10.8 years (interquartile range 7.2) were followed for a median 4.9 years (interquartile range 1.8). Overnight polysomnography evaluations at baseline health were measured and adjudicated centrally. Multivariable models created in two steps were included. First all plausible covariates were included in a screening model. Subsequently, covariates meeting significance criteria of p < 0.20 were included in the final model. Contrary to our hypothesis, higher (but not lower) mean nocturnal SpO2 was associated with higher rates of pain episodes (IRR 1.10, 95% CI [1.03-1.18], p = 0.004). Higher log OAHI did not pass screening criteria. Higher log ODI was not significantly associated with higher rates of pain episodes (IRR 0.93, 95% CI [0.82-1.06], p = 0.28). Neither low nocturnal SpO2, higher OAHI, nor higher ODI were associated with clinically relevant increased incidence rates of acute severe pain episodes. This article is protected by copyright. All rights reserved.

PMID: 29266432

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Blood. 2017 Dec 18. pii: blood-2017-06-789842. doi: 10.1182/blood-2017-06-789842. [Epub ahead of print]

Red cell exchange transfusions lower cerebral blood flow and oxygen extraction fraction in pediatric sickle cell anemia.

Guilliams KP1, Fields ME2, Ragan DK1, Eldeniz C3, Binkley MM4, Chen Y1, Comiskey LS1, Doctor A2, Hulbert ML2, Shimony JS3, Vo KD3, McKinstry RC3, An H3, Lee JM1, Ford AL5.


Blood transfusions are the mainstay of stroke prevention in pediatric sickle cell anemia (SCA), but the physiology conferring this benefit is unclear. Cerebral blood flow (CBF) and oxygen extraction fraction (OEF) are elevated in SCA, likely compensating for reduced arterial oxygen content (CaO2). We hypothesized that exchange transfusions decrease CBF and OEF by increasing CaO2, thereby relieving cerebral oxygen metabolic stress. Twenty-one children with SCA on chronic transfusion therapy (CTT) had MRIs before and after exchange transfusion. Arterial spin labeling and asymmetric spin echo sequences measured CBF and OEF, respectively, which were compared pre- and post-transfusion. Volumes of tissue with OEF above successive thresholds (36, 38, 40%), as a metric of regional metabolic stress, were compared pre- and post-transfusion. Transfusion increased Hb (9.1 to 10.3 g/dL, p<0.001) and decreased Hb S (39.7 to 24.3%, p<0.001). Transfusion reduced CBF (88 to 82.4 ml/100g/min, p=0.004) and OEF (34.4 to 31.2%, p<0.001). At all thresholds, transfusion reduced the volume of peak OEF found in the deep white matter, a location at high infarct risk in SCA (p<0.001). Reduction of elevated CBF and OEF, both globally and regionally, suggests that CTT mitigates infarct risk in pediatric SCA by relieving cerebral metabolic stress at patient and tissue-specific levels.

PMID: 29255068

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J Pediatr Hematol Oncol. 2017 Dec 15. doi: 10.1097/MPH.0000000000001052. [Epub ahead of print]

A Standardized Clinical Pathway to Decrease Hospital Admissions Among Febrile Children With Sickle Cell Disease.

Ellison AM1, Smith Whitley K, Kittick M, Schast A, Norris C, Hartung H, McKnight T, Coyne E, Lavelle J.



Recurrent hospital admissions for patients with sickle cell disease (SCD) are costly and contribute to a low quality of life for patients. We implemented a clinical pathway to safely discharge SCD patients with fever who are evaluated in the emergency department (ED) of a large tertiary care center.


An interdisciplinary team of ED and hematology physicians, nurses, and an improvement advisor developed a clinical pathway that identified febrile SCD patients at low risk of serious bacterial infection based on historical, clinical, and laboratory criteria who could be discharged from the ED. Phone follow-up was planned through the use of an automated electronic notification that was sent to an established hematology follow-up pool at the time of ED discharge. We conducted two “fake front end” trials in the ED to receive feedback on our process before full implementation. A postpathway implementation quality improvement team monitored discharge rates, phone follow-up rates and adverse events.


In the first 9 weeks postpathway implementation, 100 SCD patients were evaluated for fever; 84 (24%) met low-risk criteria and were discharged home. This reduction in admission rate has been maintained throughout the 3 years postimplementation. Successful phone follow-up was achieved in all discharged patients within 24 hours and no adverse events were identified.


Low-risk febrile patients with SCD can be safely discharged from the ED. An automated notification system within the electronic medical record system can facilitate patient follow-up after ED discharge. Future quality improvement efforts aimed to further reduce admissions in this population should target patients with modifiable risk factors for serious bacterial infection.

PMID: 29252940

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Pediatr Blood Cancer. 2017 Dec 12. doi: 10.1002/pbc.26906. [Epub ahead of print]

Parents’ pain medication underdosing is associated with more emergency department visits in sickle cell disease.

Morrison AK1, Myrvik MP2, Brousseau DC1, Drendel AL1, Scott JP2, Visotcky A3, Panepinto JA2.



To determine the association between health literacy, medication knowledge, and pain treatment skills with emergency department (ED) use of parents of children with sickle cell disease (SCD).


Parents of children 1- to 12-years-old with SCD were enrolled. Health literacy was assessed using the Newest Vital Sign. Parents completed a structured interview assessing knowledge of the dosage and frequency of home pain medications and an applied skills task requiring them to dose a prescribed pain medication. Underdosage was defined by too small a dose (dosage error) or too infrequent a dose (frequency error). The association between medication knowledge and applied skills with ED visits for pain over the past year was evaluated using Poisson regression adjusting for genotype.


One hundred parent/child pairs were included; 50% of parents had low health literacy. Low health literacy was associated with more underdose frequency errors (38% vs. 19%, P = 0.02) on the skills task. On medication knowledge, underdose dosage errors (adjusted incidence rate ratio [aIRR] 2.0, 95% confidence interval [CI] 1.3-3.0) and underdose frequency errors (aIRR, 1.7, 95% CI 1.2-2.6) were associated with a higher rate of ED visits for pain. On the skills task, underdose dosage errors (aIRR 1.6, 95% CI 1.1-2-.4) and underdose frequency errors were associated with more ED visits (aIRR 1.5, 95% CI 1.1-2.1).


For medication knowledge and skills tasks, children of parents who underdosed pain medication had a higher rate of ED visits for pain. Health literate strategies to improve parents’ medication skills may improve pain treatment at home and decrease healthcare utilization.

© 2017 Wiley Periodicals, Inc.

PMID: 29230919

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Hematology Am Soc Hematol Educ Program. 2017 Dec 8;2017(1):546-555. doi: 10.1182/asheducation-2017.1.546.

Targeting novel mechanisms of pain in sickle cell disease.

Tran H1, Gupta M2, Gupta K1.


Patients with sickle cell disease (SCD) suffer from intense pain that can start during infancy and increase in severity throughout life, leading to hospitalization and poor quality of life. A unique feature of SCD is vaso-occlusive crises (VOCs) characterized by episodic, recurrent, and unpredictable episodes of acute pain. Microvascular obstruction during a VOC leads to impaired oxygen supply to the periphery and ischemia reperfusion injury, inflammation, oxidative stress, and endothelial dysfunction, all of which may perpetuate a noxious microenvironment leading to pain. In addition to episodic acute pain, patients with SCD also report chronic pain. Current treatment of moderate to severe pain in SCD is mostly reliant upon opioids; however, long-term use of opioids is associated with multiple side effects. This review presents up-to-date developments in our understanding of the pathobiology of pain in SCD. To help focus future research efforts, major gaps in knowledge are identified regarding how sickle pathobiology evokes pain, pathways specific to chronic and acute sickle pain, perception-based targets of “top-down” mechanisms originating from the brain and neuromodulation, and how pain affects the sickle microenvironment and pathophysiology. This review also describes mechanism-based targets that may help develop novel therapeutic and/or preventive strategies to ameliorate pain in SCD.

PMID: 29222304

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Hematology Am Soc Hematol Educ Program. 2017 Dec 8;2017(1):542-545. doi: 10.1182/asheducation-2017.1.542.

Responsiveness of Patient-Reported Outcome Measurement Information System (PROMIS) pain domains and disease-specific patient-reported outcome measures in children and adults with sickle cell disease.

Curtis S1, Brandow AM2,3.


Case 1: A 33-year-old man with hemoglobin SS (homozygous hemoglobin S) disease presents for his regular clinic visit. He had 6 hospital admissions for pain over the past year. He also has avascular necrosis of the right hip. He takes daily hydroxyurea with hematologic changes indicative of compliance. He also takes morphine sustained release twice daily and morphine immediate release every 6 hours as needed for pain. He feels that more optimal pain control at home would help him reduce his number of hospital admissions in the upcoming year and improve his daily functioning at home. His hematologist decides to use Patient-Reported Outcome Measurement Information System (PROMIS) and Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-ME) to follow changes in the patient’s pain. Case 2: An 11-year-old girl with hemoglobin SS disease presents with her mother for her regular clinic visit. She had 2 admissions for pain over the past year. Her mother is concerned because she has been participating less in activities she previously enjoyed and missing classes to go to the school nurse because of pain. She is currently taking hydroxyurea and uses ibuprofen for pain. Her doctor prescribes morphine for home use but wants a way to measure if it is effective in improving her pain. Thus, her physician decides to use PROMIS and the Pediatric Quality of Life Inventory SCD (PedsQL SCD) module to determine the effectiveness of her pain control.

PMID: 29222303

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Conflict of interest statement

Conflicts of Interest: The authors declare no competing financial interests.



Hematology Am Soc Hematol Educ Program. 2017 Dec 8;2017(1):534-541. doi: 10.1182/asheducation-2017.1.534.

Pain-measurement tools in sickle cell disease: where are we now?

Darbari DS1,2, Brandow AM3,4.


Pain is a complex multidimensional experience and the most common morbidity in patients with sickle cell disease (SCD). Tools to assess pain can be of use not only to guide pain treatment but also to provide insight into underlying pain neurobiology. Mechanisms of pain in SCD are multifactorial and are not completely elucidated. Although vaso-occlusion of microcirculation by sickled red cells is believed to be the underlying mechanism of acute vaso-occlusive pain, mechanisms for chronic pain and the transition from acute to chronic pain are under investigation. A number of modalities can be used in clinical practice and/or research to capture various dimensions of pain. Selection of a pain-assessment tool should be directed by the purpose of the assessment. Pain-assessment tools, many of which are currently in the early stages of validation, are discussed here. Development and validation of these multimodal tools is crucial for developing improved understanding of SCD pain and its management.

PMID: 29222302

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Hematology Am Soc Hematol Educ Program. 2017 Dec 8;2017(1):525-533. doi: 10.1182/asheducation-2017.1.525.

Optimizing the care model for an uncomplicated acute pain episode in sickle cell disease.

Telfer P1, Kaya B2.


The pathophysiology, clinical presentation, and natural history of acute pain in sickle cell disease are unique and require a disease-centered approach that also applies general principles of acute and chronic pain management. The majority of acute pain episodes are managed at home without the need to access health care. The long-term consequences of poorly treated acute pain include chronic pain, adverse effects of chronic opioid usage, psychological maladjustment, poor quality of life, and excessive health care utilization. There is no standard protocol for management of an acute pain crisis in either the hospital or the community. The assumptions that severe acute pain must be managed in the hospital with parenteral opioids and that strong opioids are needed for home management of pain need to be questioned. Pain management in the emergency department often does not meet acceptable standards, while chronic use of strong opioids is likely to result in opioid-induced hyperalgesia, exacerbation of chronic pain symptoms, and opioid dependency. We suggest that an integrated approach is needed to control the underlying condition, modify psychological responses, optimize social support, and ensure that health care services provide safe, effective, and prompt treatment of acute pain and appropriate management of chronic pain. This integrated approach should begin at an early age and continue through the adolescent, transition, and adult phases of the care model.

PMID: 29222301

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Hematology Am Soc Hematol Educ Program. 2017 Dec 8;2017(1):440-446. doi: 10.1182/asheducation-2017.1.440.

Evolving treatment paradigms in sickle cell disease.

Jagadeeswaran R1,2, Rivers A1,2.


Sickle cell disease (SCD) is an inheritable hemoglobinopathy characterized by polymerization of hemoglobin S in red blood cells resulting in chronic hemolytic anemia, vaso-occlusive painful crisis, and multiorgan damage. In SCD, an increased reactive oxygen species (ROS) generation occurs both inside the red blood cells and inside the vascular lumen, which augment hemolysis and cellular adhesion. This review discusses the evolving body of literature on the role of ROS in the pathophysiology of SCD as well as some emerging therapeutic approaches to SCD with a focus on the reduction of ROS.

PMID: 29222291

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Hematology Am Soc Hematol Educ Program. 2017 Dec 8;2017(1):435-439. doi: 10.1182/asheducation-2017.1.435.

Chronic organ failure in adult sickle cell disease.

Vichinsky E1.


Sickle cell disease is now a chronic adult illness characterized by progressive multiorgan failure, particularly involving the brain and kidney. The etiology is multifactorial; it includes hemolysis and nitric oxide deficiency. As patients age, most experience neurologic insult. Twenty-five percent of older adults have had a clinical stroke and at least half of the population have had a silent infarct, cortical atrophy, and neurocognitive impairment. Periodic screening with neuroimaging and neurocognitive testing is recommended. Identification and correction of modifiable risk factors such as nocturnal hypoxemia, obstructive sleep apnea, and physical exercise programs should be implemented. Patients with neurocognitive impairment require cognitive remediation and educational accommodations. Chronic renal disease occurs in 25% of older adults and results in 50% of their deaths. Renal failure often develops insidiously. It can be prevented or minimized by early screening and treatment of modifiable risk factors including hypertension and microalbuminuria. There is an increasing number of therapeutic options, including inhibitors of the renin angiotensin system, angiotensin-II receptor blockers, endothelin-1 receptor antagonist, and haptoglobin therapy. Patients with sickle cell disease have increased mortality rates from renal failure compared with nonsickle cell patients, in part from a lack of access to early multidisciplinary care, including timely initiation of dialysis and renal transplantation.

PMID: 29222290

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Hematology Am Soc Hematol Educ Program. 2017 Dec 8;2017(1):431-434. doi: 10.1182/asheducation-2017.1.431.

What is the role of screening for pulmonary hypertension in adults and children with sickle cell disease?

Willen SM1, Gladwin MT2.


Patient case: An 18-year-old male patient with homozygous hemoglobin SS disease was evaluated for progressive dyspnea and elevated tricuspid regurgitant jet velocity (TRV) on echocardiography. The patient’s case is described in detail in Lancet1 He had been treated with regular transfusions since childhood for stroke, had rare episodes of vaso-occlusive pain episodes, and did not take narcotic pain medications. He presented with progressive severe dyspnea on exertion and lower extremity edema. His laboratory tests were notable for a total hemoglobin level of 11.8 g/dL and hemoglobin S levels <30% but with 18% reticulocytes and elevated markers of hemolysis, such as high plasma levels of lactate dehydrogenase, aspartate amino transferase, and indirect bilirubin. The computed tomography scan of his chest in Figure 1A-B shows a large pulmonary artery, which has a greater diameter than his aorta, and a mosaic perfusion pattern, typical for severe pulmonary arterial hypertension. His Doppler echocardiographic study (Figure 1C) showed an unusually high TRV of 5.93 m/s, consistent with a calculated pulmonary artery systolic pressure of >140 mm Hg (4 times the TRV squared = 4V2). Additional images in Figure 1D show a dilated right ventricle and right atrium with a compressed left ventricle. The patient’s right heart catheterization revealed a pulmonary artery systolic pressure of 147 mm Hg and diastolic pressure of 49 mm Hg; note that the normal values are ∼25/10 mm Hg.

PMID: 29222289

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Conflict of interest statement

Conflict-of-interest disclosure: S.M.W. has no competing financial interests. M.T.G. is on the Board of Directors or an advisory committee for Bayer; has received research funding from Bayer; holds patents with or receives royalties from the University of Pittsburgh, the National Institutes of Health, and Globin Solutions, Inc.; and has equity ownership in Globin Solutions, Inc.



Hematology Am Soc Hematol Educ Program. 2017 Dec 8;2017(1):423-430. doi: 10.1182/asheducation-2017.1.423.

Cardiovascular complications in patients with sickle cell disease.

Gladwin MT1.


Sickle cell disease (SCD) is an autosomal recessive disease in which homozygosity for a single point mutation in the gene encoding the β-globin chain produces hemoglobin S molecules that polymerize within the erythrocyte during deoxygenation; the result is sustained hemolytic anemia and vaso-occlusive events. As patients live to adulthood, the chronic impact of sustained hemolytic anemia and episodic vaso-occlusive episodes leads to progressive end-organ complications. This scenario culminates in the development of 1 or more major cardiovascular complications of SCD for which there are no approved or consensus therapies. These complications include elevated pulmonary artery systolic pressure, pulmonary hypertension, left ventricular diastolic heart disease, dysrhythmia, sudden death, and chronic kidney disease with associated proteinuria, microalbuminuria, and hemoglobinuria. In patients with advancing age, cardiopulmonary organ dysfunction and chronic kidney injury have significant effects on morbidity and premature mortality. Over the last 15 years, a number of tests have been validated in multiple replicate cohort studies that identify patients with SCD at the highest risk of experiencing pulmonary and systemic vasculopathy and death, providing for screening strategies tied to targeted, more aggressive diagnostic and therapeutic interventions.

PMID: 29222288

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Hematology Am Soc Hematol Educ Program. 2017 Dec 8;2017(1):418-422. doi: 10.1182/asheducation-2017.1.418.

Rigorous and practical quality indicators in sickle cell disease care.

Oyeku SO1, Faro EZ1.


In recent years, several sickle cell-specific quality indicators have been developed using rigorous approaches. A review of the history and current status of the development of sickle cell-specific indicators highlights opportunities for future refinement. Despite efforts at alignment, lack of strong evidence hinders the adoption of current quality indicators across stakeholder groups. There are many directions in which to take the current existing quality indicators, including expanding to different age groups, aims of care such as safety and equity, and better understanding of contextual and environmental factors.

PMID: 29222287

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Conflict of interest statement

Conflict-of-interest disclosure: The authors declare no completing financial interests.



Hematology Am Soc Hematol Educ Program. 2017 Dec 8;2017(1):412-417. doi: 10.1182/asheducation-2017.1.412.

Improving Emergency Department-Based Care of Sickle Cell Pain.

Glassberg JA1.


Pain is the leading cause of emergency department (ED) visits for individuals living with sickle cell disease (SCD). The care that is delivered in the ED is often cited by patients with SCD as the area of health care in greatest need of improvement. In 2014, the National Heart, Lung, and Blood Institute released guidelines for the care of SCD, including recommendations for the management of acute sickle cell pain in the ED. These guidelines provide a framework to understand the elements of ideal emergency sickle cell pain care; however, they do not provide guidance on barriers and facilitators to achieving these ideals in the complex system of the ED. Presented in this article are 4 tenets of implementing guideline-adherent emergency sickle cell care gleaned from the available literature and continuous quality improvement efforts at our institution. These include: (1) strategies to reduce negative provider attitudes toward patients with SCD; (2) strategies to reduce time-to-first-dose of analgesic medication; (3) strategies to improve ED pain care beyond the first dose of medication; and (4) strategies to improve ED patient safety. Application of the principles discussed within can improve patient and provider satisfaction, quality, and safety.

PMID: 29222286

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Conflict of interest statement

Conflict-of-interest disclosure: The author declares no competing financial interests.



Hematology Am Soc Hematol Educ Program. 2017 Dec 8;2017(1):406-411. doi: 10.1182/asheducation-2017.1.406.

Five lessons learned about long-term pain management in adults with sickle cell disease.

Field JJ1,2.


Chronic pain affects one-half of adults with sickle cell disease (SCD). Despite the prevalence of chronic pain, few studies have been performed to determine the best practices for this patient population. Although the pathophysiology of chronic pain in SCD may be different from other chronic pain syndromes, many of the guidelines outlined in the pain literature and elsewhere are applicable; some were consensus-adopted in the 2014 National Heart, Lung, and Blood Institute SCD Guidelines. Recommended practices, such as controlled substance agreements and monitoring of urine, may seem unnecessary or counterproductive to hematologists. After all, SCD is a severe pain disorder with a clear indication for opioids, and mistrust is already a major issue. The problem, however, is not with a particular disease but with the medicines, leading many US states to pass broad legislation in attempts to curb opioid misuse. These regulations and other key tenets of chronic pain management are not meant to deprive adults with SCD of appropriate therapies, and their implementation into hematology clinics should not affect patient-provider relationships. They simply encourage prudent prescribing practices and discourage misuse, and should be seen as an opportunity to more effectively manage our patient’s pain in the safest manner possible. In line with guideline recommendations as well as newer legislation, we present five lessons learned. These lessons form the basis for our model to manage chronic pain in adults with SCD.

PMID: 29222285

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Ann Hematol. 2017 Dec 6. doi: 10.1007/s00277-017-3199-z. [Epub ahead of print]

Sleep-disordered breathing in patients with sickle cell disease.

Raghunathan VM1, Whitesell PL2, Lim SH3,4.


Sickle cell disease is one of the most common hereditary hemoglobinopathies worldwide, and its vaso-occlusive and hemolytic crises cause considerable patient morbidity. A growing body of evidence has shown that sleep-disordered breathing, and in particular, obstructive sleep apnea, occurs at high frequency in the sickle cell population, and that there is significant overlap in the underlying pathophysiology of these two conditions. Through a variety of mechanisms including nocturnal hypoxemia and increased oxidative stress, production of pro-inflammatory cytokines, and endothelial dysfunction, sickle cell anemia and sleep-disordered breathing potentiate each other’s clinical effects and end-organ complications. Here, we will review the shared pathophysiologic mechanisms of these conditions and discuss their clinical sequelae. We will also examine the results of studies that have been carried out with clinical intervention of nocturnal hypoxemia in patients with sickle cell disease in the attempts to overcome the complications of the disease. Finally, we will propose the areas of investigation that merit further investigations in future in patients with sickle cell disease and sleep-disordered breathing.

PMID: 29214337

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Am J Hematol. 2017 Dec 5. doi: 10.1002/ajh.24994. [Epub ahead of print]

Comprehensive Management Reduces Incidence and Mortality of Acute Chest Syndrome in Patients with Sickle Cell Disease.

Basishvili G1, Gotesman J1, Vandervoort K1, Jacobs C2, Vattappally L2, Minniti CP1,2.

PMID: 29205462

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J Pediatr Hematol Oncol. 2018 Jan;40(1):51-55. doi: 10.1097/MPH.0000000000001012.

Recurrent Acute Chest Syndrome in Pediatric Sickle Cell Disease: Clinical Features and Risk Factors.

Patterson GD1, Mashegu H2, Rutherford J1, Seals S3, Josey D4, Karlson C2, McNaull M2, May W1, Carroll C1, Barr FE5, Majumdar S2.


Acute chest syndrome (ACS) is a common and serious lung complication in sickle cell disease. A retrospective medical chart review was performed over a 6-year period in all pediatric ACS patients to investigate whether factors during the initial hospitalization were associated with recurrent ACS episodes. There were 386 episodes of ACS: 149 had only 1 episode of ACS, and 76 had >1 episode of ACS; 172 (76.4%) had hemoglobin SS, and 39 (17.3%) had hemoglobin SC. The most common presenting features were fever (83%), pain (70%), and cough (61%), which changed with the number of ACS episodes. Children <4 years old were at greatest risk of recurrent ACS (P=0.018). In addition, history of asthma (adjusted incident rate ratio [IRR]=1.52; 95% confidence interval [CI], 1.22-1.98; P<0.0001), shortness of breath (IRR, 1.29; 95% CI, 1.02-1.62; P=0.033), and length of hospital stay (IRR, 1.04; 95% CI, 1.01-1.08; P=0.017) were significantly associated with prospective ACS events. Multiple episodes of ACS are common in sickle cell disease, and certain risk factors during the initial hospitalization are associated with recurrent ACS.

PMID: 29200151

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Blood. 2017 Nov 30;130(22):2377-2385. doi: 10.1182/blood-2017-05-782003.

Targeting novel mechanisms of pain in sickle cell disease.

Tran H1, Gupta M2, Gupta K1.


Patients with sickle cell disease (SCD) suffer from intense pain that can start during infancy and increase in severity throughout life, leading to hospitalization and poor quality of life. A unique feature of SCD is vaso-occlusive crises (VOCs) characterized by episodic, recurrent, and unpredictable episodes of acute pain. Microvascular obstruction during a VOC leads to impaired oxygen supply to the periphery and ischemia reperfusion injury, inflammation, oxidative stress, and endothelial dysfunction, all of which may perpetuate a noxious microenvironment leading to pain. In addition to episodic acute pain, patients with SCD also report chronic pain. Current treatment of moderate to severe pain in SCD is mostly reliant upon opioids; however, long-term use of opioids is associated with multiple side effects. This review presents up-to-date developments in our understanding of the pathobiology of pain in SCD. To help focus future research efforts, major gaps in knowledge are identified regarding how sickle pathobiology evokes pain, pathways specific to chronic and acute sickle pain, perception-based targets of “top-down” mechanisms originating from the brain and neuromodulation, and how pain affects the sickle microenvironment and pathophysiology. This review also describes mechanism-based targets that may help develop novel therapeutic and/or preventive strategies to ameliorate pain in SCD.

PMCID: PMC5709786 [Available on 2018-11-30]

PMID: 29187376 [Indexed for MEDLINE]

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Sickle Cell Conferences and Events



Community Health Worker Certification Training Program

The Sickle Cell Disease Association of America, Inc. will be hosting its third Community Health Worker Certification Training Program, with additional training on sickle cell disease. Due to circumstances beyond our control, SCDAA is postponing the start date of the Community Health Worker Training.  The training was scheduled to begin Monday, January 29, 2018.  We are rescheduling this start date to Wednesday, February 21, 2018.

If you have any questions, please contact us at

The new dates are as follows:

Date Course Title
February 21 Orientation
February 26 Primary Health Care and Human Services 1.1
February 28 Primary Health Care and Human Services 1.2
March 5 Communication
March 7 Patient Advocacy
March 12 Community Health Education
March 14 President’s Day
March 19 Capacity Building
March 21 Services Skills and Responsibilities 1.1
March 26 Services Skills and Responsibilities 1.2
March 28 SCD 101
April 2 SCD 102
April 4 Study Session
April 11 Final Exam

The seven week training will be held via webinar, Mondays and Wednesdays from 12PM-5PM EST. In addition to the online coursework, trainees must complete 80 hours of fieldwork (40 hours in a medical institution and 40 hours in a community organization). They must also pass a final exam. The training costs are estimated to be $1200 ($800 for the course plus additional fees and expenses). SCDAA now offers a Sickle Cell Disease Community Health Worker Certification Program. This one-of-a-kind certification allows individuals to learn about the fundamentals of community health workers, as well as sickle cell disease.

What is a community health worker?

According to the American Public Health Association, a community health worker (CHW) is, “A frontline public health worker who is a trusted member of and/or has an unusually close understanding of the community served.”

What qualities should a community health worker possess?

  • Connected to Community
  • Persistent, Creative and Resourceful
  • Empathic, Caring, Compassionate and Humble
  • Open-minded/Non-judgmental
  • Honest, Respectful, Patient, Realistic
  • Friendly, Engaging, Sociable
  • Dependable, Responsible, Reliable
  • Culturally sensitive, able to work with diverse communities

Who employs community health workers?

CHWs work in a variety of settings, which can include: hospitals, clinics, community-based organizations, health departments, payors, faith-based organizations, and many more.

How can community health workers help individuals with sickle cell disease?

Community health workers can assist individuals with sickle cell disease in a variety of ways. They may be asked to help identity and overcome barriers to care, such as transportation or childcare, navigate the healthcare system, assist with obtaining health insurance, conduct health education, reinforce healthy behaviors, or assist clients with other needs, as they arise.

How can I train to be a community health worker?

The Sickle Cell Disease Association of America offers a seven week sickle cell disease community health worker certification. The next training will begin on January 29, 2018. If you are interested you may download the application here

For any additional questions, please contact Jessica Suggs at or call 410-528-1555.




June 15 -17