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News

 

New information about sickle cell trait

The November 3rd issue of Annals of Internal Medicine published a comprehensive review article by leading sickle cell experts from around the US. They reviewed all of the relevant published evidence about the health outcomes of those with sickle cell trait (SCT).This review was led by the Sickle Cell Disease Association of America, the Department of Health and Human Services Secretary’s Advisory Committee on Heritable Disorders in Newborns and Children, and the American Society of Hematology to help answer many questions regarding health risks for the estimated 3.5 million Americans and 300 million with SCT worldwide.

The conclusion of the expert panel was sickle cell trait is a risk factor for increased risk of pulmonary embolism (blood clots in the legs and lung), kidney disease (Increased urine protein and chronic kidney disease), and exertional rhabdomyolysis (muscle breakdown after exertion), but does not increase the risk of  heart failure, heart attacks, stroke or decreased growth in children.  There was not enough evidence to show any increase in altitude related spleen infarction or kidney cancer (renal medullary carcinoma).

There is not enough quality published studies that show an increased risk of sudden death with extreme exercise as in the military training or athletics. A large study conducted by the Army between 2011 and 2014 showed no increase in sudden death in recruits with SCT. There have been case reports of sudden dealt in SCT athletes but no large controlled studies to determine if increased risk exists.

The authors agree with the American Society of Hematology statement recommending against routine SCT screening in athletics and support the consistent use of universal precautions to lower exertion-related risk in all persons, regardless of SCT status. There was moderate-strength evidence that SCT is a risk factor for exertional rhabdomyolysis where muscles break down, but the risk for is small, and high-intensity training and genetics may play a role

The increased risk for forming blood clots among SCT carriers compared with noncarriers is at a level similar to that found in other low-risk genetic causes of clotting that do not require screening.

This review helped to answer many questions about SCT carriers, however it pointed out the need for more well designed, controlled studies to determine the health risks and methods to lower those risks.

Naik RP, Smith-Whitley K, Hassell KL, Umeh NI, de Montalembert M, Sahota P, et al. Clinical Outcomes Associated With Sickle Cell Trait: A Systematic Review. Ann Intern Med. ;169:619–627. doi: 10.7326/M18-1161

 

 

Study affirms challenges in managing severe pain of sickle cell disease

Findings underscore need to find alternatives and supplements to opioids, researchers say https://www.eurekalert.org/pub_releases/2018-11/jhm-sac112618.php

In a study tracking the severe crisis pain of sickle cell disease and its management in 73 adults over a period of a year, Johns Hopkins researchers found that even among those on high doses of daily at-home opioids, a persistent subset was more likely to seek emergency hospital care for crisis pain and was less likely to have the pain controlled by intensive treatment.

The researchers say their findings, described in the September issue of the American Journal of Hematology, underscore the persistent difficulties, poor patient outcomes and high costs associated with assessing and addressing the 10 to 20 percent of patients with sickle cell who are the sickest and have the most pain.

“Although progress has been made in managing the pain crises of many with sickle cell, there remains a group of sicker patients who seek hospital care with greater than typical frequency and whose pain is not being treated effectively,” says C. Patrick Carroll, M.D., director of psychiatric services for the Johns Hopkins Sickle Cell Center for Adults and assistant professor of psychiatry and behavioral sciences at the Johns Hopkins University School of Medicine. “We want to focus our efforts on figuring out how to deliver high value care to our sickest patients.”

Sickle cell disease is the most common inherited blood disorder diagnosed in the United States, affecting an estimated 100,000 people, most of them African-Americans. In addition, about one in 13 Americans of African descent carry one copy of the gene that causes sickle cell disease, and have “sickle cell trait.” People who inherit two copies have sickle cell anemia, the disorder’s most common form. The disorder is marked by the characteristic “sickled” or crescent-shape red blood cells that can get stuck in small blood vessels feeding bones, creating recurrent bouts of crippling pain that require opioids and sometimes urgent hospitalization. Beyond the disabling toll on patients, the disease accounts for a significant amount of health care costs — an estimated $500 million per year. About 10-20 percent of people with sickle cell account for more than 50 percent of the costs, Carroll says, reflecting the reality of patients with sickle cell whose pain episodes are more frequent and more intense than usual.

“The most clinically interesting finding but also the most puzzling was the extent to which higher opioid doses — both at home and during acute visits — were tied to poorer outcomes and more complications,” says Carroll. “There is the conundrum that despite more aggressive treatment, a subset of people didn’t get as much benefit.”

He says growing tolerance to opioids may be one explanation, along with emergency room physicians who don’t know a patient’s history to quickly provide adequate pain medicines when pain crises occur, which may require higher opioid doses than are safe for a typical patient. Care is fragmented, Carroll says, and because there are no objective measures of pain, some physicians are reluctant to prescribe higher doses of opioids.

In their study that documented the source of higher-than-typical infusion center visits, the researchers looked at data from 73 patients seen at the Johns Hopkins Sickle Cell Center for Adults. Patients were an average age of 34, and 62 percent were women. Participants all underwent assessment on a standard Pain Anxiety Symptoms Scale, and researchers collected information on patients’ socioeconomic status, insurance coverage and education level. The researchers relied on medical records to document admission to the Sickle Cell Infusion Center, where patients get treated for crisis pain. Opioid doses were converted to a standard measurement of “morphine equivalents” so drug quantity could be easily compared among participants.

With those data, the researchers classified 23 people as “typical” users of the infusion center (less than five visits over a year). Another 23 people were considered “high” users of the infusion center (five visits or more). The remaining 27 people had no visits in a calendar year.

Typical users of the infusion center were on an average of about 26 morphine equivalents of opioids daily at home, compared to high-users who were on about 66 morphine equivalents of opioid medication daily.

Although the typical users had on average the same initial crisis pain rating as the high-users (8.5 versus 8.4 on a scale of 10), the typical users of infusion center care reported an average reduction of 3.8 pain points after treatment with opioids intravenously, putting their pain level around 5, compared to the high-users of infusion center care who only reported a drop of an average of 1.6 pain points, putting them at around 7 for reported pain after treatment. Pain improvement was twice as great for typical infusion center users, yet they received less than half the opioid dosage (~26 milligrams) during the emergency visits than those high users of the infusion center (~66 milligrams).

Because it’s a challenge to manage pain effectively without prescribing potentially unsafe amounts of opioids, what’s clear, Carroll says, is the need to develop more nonopioid pain relievers that don’t increase risks of tolerance and overdose.

One of the biggest drivers of cost and ineffective treatment of people with sickle cell, he says, is that in many cases the health care team dealing with people in an emergency setting during crisis isn’t the same providers who help the patient manage day-to-day care. “This typically means that emergency care providers don’t reliably know medication dosages and treatment plans in place for that person,” Carroll says.

There is a great need, he adds, for sickle cell disease clinical centers that manage both day-to-day and 24/7 emergency care, such as those with the integrated approach used by the Johns Hopkins Sickle Cell Infusion Center that can help bridge the care gap and keep treatment consistent.

 

 

New Resources

Novartis is inviting nonprofit organizations to submit ideas for potential funding through the 2018 STEP (Solutions to Empower Patients) Program™. The Program will recognize and fund as many as five proposals of up to $50,000 each that demonstrate innovation in health education, empowerment, and support to help improve the experience of people living with SCD. Proposals will be evaluated by an external review committee consisting of experts in a variety of fields including advocacy, psycho-social support, multi-cultural health and a SCD practitioner and patient.

You can find more information about the Program and submission process in the release below and here.

Novartis to fund five innovative ideas to support patients and the sickle cell community

  • 2018 STEP Program™ designed to support unique proposals from nonprofit organizations that serve people living with sickle cell disease
  • Novartis will select proposals that demonstrate innovation in health education and support to improve the experience of patients and their families

Novartis announced the launch of the 2018 STEP (Solutions to Empower Patients) Program™ to inspire solutions that support people living with sickle cell disease (SCD).

The program will recognize and fund as many as five proposals of up to $50,000 each that demonstrate innovation in health education, empowerment and support to help improve the experience of people living with SCD. In 2017, the first year of the initiative, the STEP Program provided funding to three organizations involved with metastatic breast cancer.

“People living with sickle cell disease face unique challenges,” said Ameet Mallik, Executive Vice President and Head, US, Novartis Oncology. “By helping fund innovative programs developed by the organizations that best understand these challenges, we hope to make the biggest impact for those affected by this disease, where there continues to be a significant unmet need.”

Application Details for Interested Health Care or Health Care-Related Nonprofit Organizations

Any US-based, 501(c)(3) organization that supports people impacted by SCD is welcome to submit a STEP Program proposal for consideration. Suggested proposals should focus on at least one of the following:

  • Empowering SCD patients to advocate for themselves
  • Providing resources, programs or tools that help SCD patients better interact with health care providers (HCPs) and navigate the health care system
  • Educating HCPs about SCD management and patient needs
  • Providing innovative programs to ease the transition to adult care
  • Providing training programs for HCPs and raising awareness of implicit and institutional biases
  • Proposals will be evaluated by an external review committee consisting of experts in a variety of fields. The committee will review the applications and evaluate and identify as many as five proposals on the basis of innovation and the potential to make the greatest positive impact on the SCD community.

Organizations that wish to apply for funding from the STEP Program should send their completed submission form to STEP.Program@Novartis.com. All submissions must be received no later than December 21, 2018.

For more information about the STEP Program, please see the program backgrounder and submission FAQ.

 

 

New ResourcesStride 2 Study Large MultiCenter Study in the US and Canada

BMT CTN 1503 (STRIDE 2) is a clinical trial to find out if bone marrow transplantation is better than standard of care in improving survival and decreasing sickle cell-related complications for young adults with severe SCD. To understand which treatment option is most effective, we need a side-by-side comparison of bone marrow transplant and standard of care for SCD. BMT CTN 1503 (STRIDE 2) is the first trial to compare these treatment options for SCD. Currently, we do not know if bone marrow transplants is better than the standard of care for treating SCD in adults. The trial is open in 46 centers in the United States and Canada and will enroll 200 patients. Participation in this trial is voluntary.

What is the study process?

Interested participants will be screened for eligibility. If you are eligible for the study, you will be tissue typed (HLA-typed) and will undergo a donor search to determine if a suitable related or unrelated donor is available. If you have a donor match, you will be assigned to the donor arm of the study and will undergo a bone marrow transplant. If you do not have a suitable donor match, then you will be assigned to the no donor arm of the study and will continue with your usual standard of care treatment.

Are you eligible?

  • Do you have severe sickle cell disease?
  • Are you 15-40 years of age?
  • Have you ever had a stroke or serious brain complication?
  • Have you had two or more episodes of acute chest syndrome in the last 2 years?
  • Have you had three of more episodes of severe pain crises in the last 2 years?
  • Have you received eight or more blood transfusions in the last year?
  • Do you have a history of an abnormal echocardiogram?

For more information, please click on the following link to see the Stride 2 website: https://jarushealthcloud.emory.edu/StrideApplication/

Making difficult decisions regarding sickle cell disease can be hard. For help deciding next steps, please feel free to use the Decision-Aid website for more information:

www.sickleoptions.org

If you are interested in speaking to a doctor about possible enrollment, please reach out to Shannon Smith at shannon.l.smith@emory.edu or 404-785-4389

For more info about the Decision Aid website, please reach out to Diana Ross at diana.ross@emory.edu or 404-727-978

 

 

New Books

Dr. Ali AL-Jama and Dr. Ibrahim Al-Dabbous second edition book “Management Manual of Sickle Cell Disease”, is an updated comprehensive and evidence based guide for clinicians in the Middle East. This is a practical and well organized  guide that all providers in the region should use to deliver excellent care to the sickle cell population the book can be obtained by contacting DanaAlmaarif@hotmail.com.

New Link for the Book A Doctor In A Patient’s Body; Dreaming Big with Sickle Cell Disease and Chronic Pain.” by  Dr. Simone Eastman Uwan  at https://www.amazon.com/dp/1731044267

 

 

Articles in the Medical Literature

 

Clin Neuropsychol. 2018 Nov 24:1-17. doi: 10.1080/13854046.2018.1535664. [Epub ahead of print]

Integration of neuropsychology services in a sickle cell clinic and subsequent healthcare use for pain crises.

Janecek JK1, Dorociak KE2, Piper LE3, Kelleher T4, Pliskin NH2, Gowhari M5, Molokie RE5,6.

Abstract

OBJECTIVE:

Growing literature has documented the clinical utility of neuropsychological evaluations for predicting functional outcomes, including reduced healthcare service utilization, in a variety of clinical samples. The present study investigates the relationship between the integration of clinical neuropsychology services into an existing outpatient sickle cell clinic and frequency of emergency department (ED) visits and hospitalizations for pain crises.

METHOD:

Participants included 144 adults diagnosed with sickle cell disease (SCD) who either underwent neuropsychological evaluation (NP+), including interview, neuropsychological testing, and feedback, or treatment as usual (NP-). Medical records were reviewed for a two-year period, one year prior to study enrollment (pre-assessment) and one year post-study enrollment (post-assessment), to track the number of ED visits and hospitalizations related to sickle cell pain crises.

RESULTS:

When examining pain crises ED visits prior to and following neuropsychological evaluation, there was a significant decrease in ED visits for the NP + group, but no change for the NP - group. No significant changes in pain crises hospitalizations were observed for the NP + and NP - groups. For the NP + group, the decreased incidence of pain crises ED visits and hospitalizations was associated with an estimated total cost savings of $994,821.

DISCUSSION:

Results highlight that integration of neuropsychology services into an existing outpatient sickle cell clinic may reduce healthcare costs, particularly use of pain crises ED services, for adults with SCD.

PMID: 30472925

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Br J Haematol. 2018 Nov 21. doi: 10.1111/bjh.15651. [Epub ahead of print]

Effect of renin-angiotensin-aldosterone system blocking agents on progression of glomerulopathy in sickle cell disease.

Thrower A1, Ciccone EJ2, Maitra P3, Derebail VK4, Cai J3, Ataga KI5.

Abstract

Although renin-angiotensin-aldosterone system (RAAS) blocking agents decrease albuminuria in short-term studies, there is no evidence confirming their long-term efficacy in sickle cell disease (SCD). In a single-centre, retrospective study, we evaluated the long-term effect of RAAS blocking agents on proteinuria and declining estimated glomerular filtration rates (eGFR). Eighty-six patients on RAAS blocking agents for proteinuria, followed for a median of 2·28 years, were compared with 68 patients with proteinuria followed for 2·24 years who were not receiving such treatment. The log odds of proteinuria decreased over time in patients on RAAS blocking agents (β: -0·23, P = 0·03) and in the non-treatment group (β: -0·54, P < 0·0001), but was not statistically different between both groups (β: 0·31, P = 0·063). The eGFR declined over time in patients on RAAS blocking agents (β: -2·78, P < 0·0001) and in those not on such treatment (β: -4·7, P < 0·0001), and was statistically different between both groups (β: 1·9, P = 0·0002). Baseline eGFR was associated with mortality (Hazard rato: 0·97, P < 0·0001), but RAAS blocking agents had no significant effect on mortality. These data suggest that RAAS blockade may slow the loss of kidney function in SCD.

PMID: 30460977

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Am J Hematol. 2018 Nov 19. doi: 10.1002/ajh.25356. [Epub ahead of print]

Multidisciplinary care results in similar maternal and perinatal mortality rates for women with and without SCD in a low-resource setting.

Oppong SA1,2, Asare EV3,4, Olayemi E3,5, Boafor T1,2, Dei-Adomakoh Y3,5, Swarry-Deen A2, Mensah E5, Osei-Bonsu Y3, Crabbe S3, Musah L6, Hayfron-Benjamin C6,7, Covert B8, Kassim AA8, James A9, Rodeghier M10, Audet C11, DeBaun MR8.

Abstract

In Africa, the maternal mortality rate in sickle cell disease (SCD) is approximately 10%. Our team previously demonstrated an 89% decrease in mortality rate in a before-and-after feasibility study among women with SCD living in low-resource setting in Ghana. In the same cohort including additional participants with and without SCD, we used a prospective cohort design to test the hypothesis that implementing a multidisciplinary care team for pregnant women with SCD in low-resource setting will result in similar maternal and perinatal mortality rates compared to women without SCD. We prospectively enrolled pregnant women with and without SCD or trait and followed them up for six-week postpartum. We tested the newborns of mothers with SCD for SCD. We recruited age and parity matched pregnant women without SCD or trait as the comparison group. Maternal and perinatal mortality rates were the primary outcomes. A total of 149 pregnant women with SCD (HbSS, 54; HbSC, 95) and 117 pregnant women without SCD or trait were included in the analysis. Post-intervention, maternal mortality rates were 1.3% and 0.9% in women with and without SCD, respectively (p = 1.00); the perinatal mortality rates were 7.4% and 3.4% for women with and without SCD, respectively (p = 0.164). Among the mothers with SCD, approximately 15% of newborns had SCD. Multidisciplinary care of pregnant women with SCD may reduce maternal and perinatal mortality rates to similar levels in pregnant women without SCD in low-resource settings. Newborns of mothers with SCD have a high rate of SCD. This article is protected by copyright. All rights reserved.

PMID: 30456766

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Br J Haematol. 2018 Nov 16. doi: 10.1111/bjh.15646. [Epub ahead of print]

Ticagrelor does not impact patient-reported pain in young adults with sickle cell disease: a multicentre, randomised phase IIb study.

Kanter J1, Abboud MR2, Kaya B3, Nduba V4, Amilon C5, Gottfridsson C6, Rensfeldt M7, Leonsson-Zachrisson M7; HESTIA2 study investigators.

Abstract

Ticagrelor is an antiplatelet agent for adults with coronary artery disease. The inhibition of platelet activation may decrease the frequency of vaso-occlusion crisis (VOC) in sickle cell disease (SCD). The HESTIA2 study (NCT02482298) randomised 87 adults with SCD (aged 18-30 years) 1:1:1 to twice-daily ticagrelor 10, 45 mg or placebo for 12 weeks. Numerical decreases from baseline in mean proportion of days with patient-reported pain (primary endpoint) were seen in all three groups, as well as in pain intensity and analgesic use, with no significant differences between placebo and ticagrelor treatment groups. Plasma ticagrelor concentrations and platelet inhibition increased with dose. Adverse events were distributed evenly across groups and two non-major bleeding events occurred per group. Ticagrelor was well tolerated with a low bleeding risk, but no effect on diary-reported pain was detected. Potential effects on frequency of VOCs will need to be evaluated in a larger and longer study.

PMID: 30443999

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Mayo Clin Proc. 2018 Nov 7. pii: S0025-6196(18)30585-8. doi: 10.1016/j.mayocp.2018.08.001. [Epub ahead of print]

Advances in the Treatment of Sickle Cell Disease.

Kapoor S1, Little JA1, Pecker LH2.

Abstract

Sickle cell disease (SCD) is a monogenic disorder that afflicts approximately 100,000 Americans and millions of people worldwide. It is characterized by hemolytic anemia, vaso-occlusive crises, relentless end-organ injury, and premature death. Currently, red blood cell transfusion and hydroxyurea are the major disease-modifying therapies available for SCD. Hematopoetic stem cell transplant is curative, but barriers to treatment are substantial and include a lack of suitable donors, immunologic transplant rejection, long-term adverse effects, prognostic uncertainty, and poor end-organ function, which is especially problematic for older patients. Gene therapy to correct the βs point mutation is under investigation as another curative modality. Deeper insights into the pathophysiology of SCD have led to the development of novel agents that target cellular adhesion, inflammation, oxidant injury, platelets and/or coagulation, vascular tone, and hemoglobin polymerization. These agents are in preclinical and clinical trials. One such agent, L-glutamine, decreases red blood cell oxidant injury and is recently US Food and Drug Administration approved to prevent acute pain episodes of SCD in patients 5 years of age or older. The purpose of this review is to describe the currently established therapies, barriers to curative therapies, and novel therapeutic agents that can target sickle cell hemoglobin polymerization and/or its downstream sequelae. A PubMed search was conducted for articles published up to May 15, 2018, using the search terms sickle cell disease, novel treatments, hematopoietic stem cell transplantation, and gene therapy. Studies cited include case series, retrospective studies, prospective clinical trials, meta-analyses, online abstracts, and original reviews.

PMID: 30414734

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Pediatr Blood Cancer. 2018 Nov 4:e27538. doi: 10.1002/pbc.27538. [Epub ahead of print]

Pediatric pain screening identifies youth at risk of chronic pain in sickle cell disease.

Sil S1,2, Cohen LL2,3, Dampier C1,2.

Abstract

BACKGROUND:

This study aimed to evaluate the preliminary validation and application of a pain screening tool to identify biopsychosocial risk factors for chronic pain in pediatric sickle cell disease (SCD) and classify youth with SCD into prognostic risk groups.

METHOD:

Youth presenting to a pediatric SCD clinic completed the Pediatric Pain Screening Tool (PPST), a brief 9-item self-report questionnaire developed for rapid identification of risk in youth with pain complaints. Youth also completed a battery of standardized patient-reported outcomes, including pain characteristics, pain burden, functional disability, pain interference, depressive symptoms, pain catastrophizing, and fear of pain. Healthcare utilization was extracted from medical chart review.

RESULTS:

Seventy-three 8- to 18-year-olds (94% Black, 57% female) with SCD participated. The PPST demonstrated discriminant validity that ranged from fair to excellent (area under the curves (AUC) = 0.74-0.93, P values < 0.001) for identifying significant pain frequency, disability, pain interference, and psychosocial distress. Receiver operating characteristic curve analyses indicated that previously established cutoff scores were appropriate for the SCD sample. Participants were classified into low-risk (28.8%), medium-risk (38.4%), and high-risk (32.9%) groups, with significant group differences across measures, F(18, 116) = 6.67, P < 0.001. The high-risk group reported significantly higher pain intensity, pain frequency, pain burden, functional disability, pain interference, and depressive symptoms relative to both low-risk and medium-risk groups (P values < 0.005).

CONCLUSIONS:

The high-risk group demonstrated a pain and psychosocial profile consistent with chronic SCD pain. The PPST may be useful for efficiently identifying youth with chronic SCD pain or those at risk of poor outcomes.

© 2018 Wiley Periodicals, Inc.

PMID: 30393948

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Blood Cells Mol Dis. 2019 Feb;74:25-29. doi: 10.1016/j.bcmd.2018.10.005. Epub 2018 Oct 22.

Metabolic syndrome among adults living with sickle cell disease.

Ogunsile FJ1, Bediako SM2, Nelson J3, Cichowitz C4, Yu T5, Patrick Carroll C4, Stewart K4, Naik R4, Haywood C Jr4, Lanzkron S4.

Abstract

Metabolic syndrome (MetS) is a key risk factor for cardiovascular disease (CVD) incidence and all-cause mortality. MetS prevalence among adults with sickle cell disease (SCD) is not well known. We report initial findings from a cross-sectional study that examined MetS risk factors within a cohort of adults living with SCD. 50 adult SCD participants (ages 21-66 years; 72% female) completed demographic and health behavior surveys, health-related family and personal histories, and anthropometric and laboratory measurements. Descriptive and inferential statistics were used to summarize and compare CVD risk factors, stratified in separate analyses by SCD genotype and sex. Triglyceride, blood pressure, and fasting glucose levels were within normal limits. 78% of the cohort reported moderate to high physical activity. However, 46% of this cohort was overweight and dietary saturated fat intake exceeded both the national average (11%) and US Dietary Guidelines (<10%). 14.3% of the cohort fulfilled criteria for MetS with large waist circumference and reduced HDL levels prominently accounting for this status. We evaluated the prevalence of MetS in a cohort of adults living with SCD. Our findings suggest that increased attention to eating habits and physical activity may generate new approaches for decreasing cardiovascular morbidity in SCD.

PMID: 30391047

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Lancet Haematol. 2018 Nov;5(11):e554-e562. doi: 10.1016/S2352-3026(18)30163-7.

Moderate-intensity endurance-exercise training in patients with sickle-cell disease without severe chronic complications (EXDRE): an open-label randomised controlled trial.

Gellen B1, Messonnier LA2, Galactéros F3, Audureau E4, Merlet AN5, Rupp T2, Peyrot S6, Martin C7, Féasson L8, Bartolucci P9; EXDRE collaborative study group.

Collaborators: (16)

Habibi A, Guillet E, Gellen-Dautremer J, Ribeil JA, Arlet JB, Mattioni S, Berkenou J, Delrieux N, Lionnet F, Grenot JF, Mira J, Peyrard A, Lacroix R, Garcin A, Di Liberto G, Hourdé C.

Abstract

BACKGROUND:

Exercise could be a triggering factor for vaso-occlusive crises in patients with sickle-cell disease. We aimed to investigate whether a patient-adapted training programme of moderate endurance exercise could be safe and beneficial for patients with sickle-cell disease.

METHODS:

We did a multicentre, prospective, open-label, randomised controlled trial at four university hospitals in France. Eligible patients were older than 18 years, with an HbSS or S/β0-thalassaemia genotype, and with no severe chronic complications. All patients underwent cardiopulmonary exercise tests (CPETs) on a stationary bicycle with cardiac, pulmonary, laboratory, and muscle parameter evaluations at the start and end of the study period. We randomly assigned patients (1:1) to the training group (three 45-min exercise sessions per week, for 8 weeks) or the control group (no lifestyle changes) using a central computer-generated randomisation list. During baseline evaluation, patients and researchers were masked to group assignment; randomisation was done after completion of the baseline evaluation to minimise bias. The primary outcome was difference in power output at a blood lactate concentration of 4 mmol/L during CPET between baseline and the end of the 8-week training period. Patients were analysed on a per-protocol basis, excluding those who missed more than 20% of the training sessions or had other major protocol violations. This trial is registered with ClinicalTrials.gov, number NCT02571088, and is completed.

FINDINGS:

Between Sept 8, 2014, and Dec 11, 2015, 40 patients were enrolled (20 to each group). After exclusion of seven patients (one pregnancy, one appendicitis, one protocol violation, two lost to follow-up, and two incompatibilities with work schedule), 33 patients were analysed (15 in the training group, 18 in the control group). At the 8-week follow-up, the absolute change from baseline in mean power output at 4 mmol/L blood lactate was 7·2 W (SD 8·7) in trained patients (from 70·4 W [SD 16·2] at baseline to 77·6 W [15·1] at end of intervention) compared with -0·3 W (9·4) in controls (from 66·2 W [13·8] to 65·9 W [15·6]; mean difference between groups 7·3 W [95% CI 0·7-13·8], p=0·031). No adverse events requiring hospital admission occurred in the training group, whereas five occurred in the control group: four (20%) vaso-occlusive crises (one complicated by an acute chest syndrome), and one (6%) viral infection with isolated chest pain (hazard ratio 0·143 [95% CI 0·024-0·827; p=0·029).

INTERPRETATION:

Moderate-intensity endurance-exercise training seems to be safe for adults with sickle-cell disease without severe chronic complications and significantly improved their functional capacity, especially for exercise levels close to those needed for daily activities. Our findings support consideration of endurance-exercise training as a novel therapeutic strategy for patients with sickle-cell disease.

FUNDING:

Société Française de Cardiologie and Institut National du Sport, de l’Expertise et de la Performance (INSEP).

Copyright © 2018 Elsevier Ltd. All rights reserved.

PMID: 30389037

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Pediatr Blood Cancer. 2018 Nov 1:e27535. doi: 10.1002/pbc.27535. [Epub ahead of print]

Pediatric residents’ perceived barriers to opioid use in sickle cell disease pain management.

Fearon A1, Marsh A2, Kim J2, Treadwell M2.

Abstract

OBJECTIVE:

Current guidelines recommend high-priority treatment of severe sickle cell disease (SCD) pain with opioids; however, patients with SCD have historically been undertreated. We used mixed methods to assess pediatric residents’ perceptions toward opioid use in SCD pain management.

METHODS:

We distributed a survey to 88 residents at an urban pediatric medical center in a cross-sectional study. Participants responded to questions about perceived barriers to acute SCD pain management and attitudes toward patients with SCD. Responses were examined using bivariate analyses. Five pediatric residents were interviewed, to provide more in-depth understanding of barriers to SCD pain management.

RESULTS:

Fifty-three residents (60%) completed the survey. Participants were divided into “more experienced” (had seen ≥ 21 patients with SCD; 45.3%) or “less experienced.” Both groups reported potential for tolerance and dependence as major barriers to opioid use in SCD. Less experienced residents reported a greater need for additional training in SCD pain management (P < 0.05), more concern about addiction, and greater empathy for patients with SCD (P < 0.05). Both groups agreed that patients with SCD were “frustrating to care for.” Thematic analysis revealed that increased patient and provider barriers led to distrust, ultimately leading to undertreatment of pain and inadequate care.

CONCLUSION:

Although more experienced residents reported feeling more comfortable treating acute SCD pain and were less concerned with addiction compared with less experienced residents, certain negative views of patients with SCD were prevalent among all residents. Findings suggest that residency training must address provider attitudes as well as knowledge about SCD.

© 2018 Wiley Periodicals, Inc.

PMID: 30387290

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Pediatr Blood Cancer. 2018 Nov 1:e27507. doi: 10.1002/pbc.27507. [Epub ahead of print]

Student perspectives on managing sickle cell disease at school.

Haridasa N1, DeBaun MR1, Sanger M2, Mayo-Gamble TL3.

Abstract

OBJECTIVE:

The study objective was to identify the perceptions of children with sickle cell disease (SCD) in the school environment.

METHODS:

Semistructured interviews (N = 14) were conducted with pediatric patients ages 6 to 10 who attended Metro Nashville Public Schools. These participants were recruited through the Vanderbilt Sickle Cell Disease Clinic. Participants were asked about the perceived efficacy of their teachers to (1) understand SCD; (2) communicate with students regarding SCD; (3) handle an SCD-related pain episode in school; and (4) identify methods to compensate for school absenteeism associated with an SCD diagnosis. Content analysis identified underlying themes.

RESULTS:

Five themes emerged that highlighted the perceptions and recommendations of children with SCD in the school environment: (1) perceptions that allow students to prevent SCD from limiting their school experience; (2) administrator actions to alleviate challenges associated with SCD; (3) communication about SCD; (4) how SCD interferes with school activities; and (5) ways students advocate for themselves. Students also provided four areas of recommendations for school personnel: (1) ways teachers can help with school activities; (2) make-up work for school absences; (3) empowering students with SCD; and (4) helping with SCD episodes at school.

CONCLUSIONS:

Students with SCD advocated strongly for their needs at school to attain their education. However, they perceived school personnel to lack knowledge about SCD management. This could be overcome with a handbook specific for teachers of students with SCD that could address each of the five themes.

© 2018 Wiley Periodicals, Inc.

PMID: 30387236

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Sickle Cell Conferences and Events

 

Old and New Treatments for Sickle Cell Anemia: Is There a Standard of Care?

Presented by Cincinnati Children’s Hospital Medical Center

Friday, November 30, 2018 12:30-4:30 pm PST

Marriott Marquis San Diego Marina

333 West Harbor Drive, Marina Ballroom D

San Diego, CA 92101

Target audience:

Hematologists and other healthcare professionals involved in the treatment of patients with sickle cell disease.

Overview:

Over the past few years, a plethora of new therapeutic options has emerged for sickle cell anemia, some to prevent or ameliorate acute complications and others designed to reduce sickling and hemolytic anemia. How these new therapies fit into current and future treatment paradigms remains unclear. This exciting symposium will bring together leading experts to discuss both old and new treatments for sickle cell anemia. Faculty members from high-resource and low-resource countries will provide their perspectives about what represents standard of care for sickle cell anemia, where the current treatment gaps and challenges lie, and how clinicians can determine the best therapeutic choices for their patients.

Learning Objectives:

Upon completion of this activity, participants should be better able to:

  1. Describe established and FDA-approved treatments for sickle cell anemia
  2. Delineate barriers to treatment, particularly among adults with sickle cell anemia
  3. Describe treatment options for pregnant and lactating women
  4. Outline new and emerging therapies for sickle cell anemia
  5. Explain treatment options in low-resource settings including Sub-Saharan Africa

REGISTER NOW: at https://sicklecellfss.eventbrite.com

 

 

‘Making Hydroxyurea Therapy Available and Affordable in Low-resource Settings’

at the Annual American Society of Hematology (ASH) meeting is taking place on Sunday, December 2, 2018 from 11:15 to 12:30pm in Marina Ballroom F.