News
Crizanlizumab for the Prevention of Pain Crises in Sickle Cell Disease
http://www.nejm.org/doi/full/10.1056/NEJMoa1611770#t=article
The up-regulation of P-selectin in endothelial cells and platelets contributes to the cell–cell interactions that are involved in the pathogenesis of vaso-occlusion and sickle cell–related pain crises. The safety and efficacy of crizanlizumab, an antibody against the adhesion molecule P-selectin, were evaluated in patients with sickle cell disease.In patients with sickle cell disease, crizanlizumab therapy resulted in a significantly lower rate of sickle cell–related pain crises than placebo and was associated with a low incidence of adverse events. (Funded by Selexys Pharmaceuticals and others; SUSTAIN ClinicalTrials.gov)
SUSTAIN: A Multicenter, Randomized, Placebo-Controlled, Double-Blind, 12-Month Study to Assess Safety and Efficacy of SelG1 with or without Hydroxyurea Therapy in Sickle Cell Disease Patients with Sickle Cell-Related Pain Crises
https://ash.confex.com/ash/2016/webprogram/Paper92707.html
Sickle Cell Disease Coalition
http://www.scdcoalition.org/sickle-cell-disease.html
People with sickle cell disease (SCD) are afflicted on two fronts — one by having a serious, chronic condition that inflicts pain and other complications — the other by a fragmented system of care.
Today, there are opportunities to transform this disease and the way we care for people with SCD. We are launching an international call to action on SCD by bringing together researchers, clinicians, individuals with sickle cell disease and their families, policymakers, and the private sector to focus our collective efforts and change the state of SCD around the world
ASH report on the state of Sickle Cell Disease
http://www.scdcoalition.org/report.html
The State of Sickle Cell Disease: 2016 Report has been created by the American Society of Hematology (ASH) based on the feedback of more than 100 thought leaders and representatives from other organizations to look at the state of SCD and identify the greatest opportunities for improvement. This report is a summary of the current state of research, access to care, and global issues, and it provides an outline of opportunities to change the status quo for people with this disease.
Selected December American Society of Hematology (ASH) Sickle Cell Abstracts
Control/Click on a number to see the abstract
Sickle 266, 313, 319, 858, 1319, 2485, 3639
Sickle Cell 158, 202, 265, 1294, 1302, 1310, 1321, 1322, 1323, 2340, 2449, 2475, 2484, 2487, 2490, 2491, 2499, 3531, 3534, 3536, 3591, 3644, 3652, 3654, 3668, 3669, 3670, 3673, 3674
Sickle Cell Adhesion 1311, 2482, 2509, 3643
Sickle Cell Anemia 8, 10, 125, 853, 854, 1015, 1018, 1020, 1233, 1257, 1298, 1311, 1320, 2331, 2390, 2446, 2465, 2479, 2494, 2496, 3468, 3637, 3643, 3653, 3657, 3658, 3667, 3839, 3842, 4586
Sickle Cell Disease 1, 7, 9, 11, 12, 95, 121, 122, 123, 124, 126, 160, 161, 162, 247, 248, 267, 268, 269, 315, 316, 317, 318, 324, 855, 857, 1016, 1017, 1245, 1274, 1276, 1292, 1293, 1295, 1296, 1297, 1299, 1300, 1301, 1304, 1305, 1306, 1307, 1308, 1309, 1312, 1313, 1314, 1315, 1316, 1317, 1318, 1323, 1324, 1351, 2311, 2332, 2344, 2390, 2392, 2393, 2472, 2473, 2474, 2476, 2477, 2478, 2480, 2481, 2482, 2483, 2486, 2487, 2488, 2489, 2492, 2493, 2495, 2497, 2498, 2500, 2501, 3404, 3525, 3526, 3529, 3532, 3539, 3589, 3629, 3634, 3635, 3638, 3640, 3642, 3645, 3646, 3647, 3648, 3649, 3650, 3651, 3655, 3656, 3659, 3660, 3661, 3662, 3663, 3664, 3665, 3666, 3671, 3672, 3716, 3809, 3814, 3856, 4703, 4706, 4747, 4749, 4757, 4760, 4763
Sickle Cell Patient 4744
Mortality Plummets in Pregnant Women With Sickle Cell Disease
A multidisciplinary approach dramatically reduced mortality in pregnant women with sickle cell disease (SCD), after being in place for only 13 months.
The Korle-Bu Teaching Hospital, a national referral center in Accra, Ghana, treats approximately 250 pregnant women with SCD every year; up to 12% die during pregnancy and after childbirth.
But after the intervention, there was an 89% decline in maternal deaths (9.5% vs 1.1%) along with a 62% reduction in perinatal mortality (60.8% vs 23% per 1000 total births).
The findings were presented here at the American Society of Hematology (ASH) 2016 Annual Meeting (abstract 1017). http://www.medscape.com/viewarticle/872788
Case series of octogenarians with sickle cell disease
Samir K. Ballas, E. Dianne Pulte, Clarisse Lobo and Gaye Riddick-Burden
Blood 2016 128:2367-2369; doi: http://dx.doi.org/10.1182/blood-2016-05-715946 http://www.bloodjournal.org/content/128/19/2367
Survival of patients with sickle cell disease (SCD) has increased progressively since the 1980s. Patients with SCD in their sixth or seventh decade have been described previously. This report describes 4 patients with SCD who survived beyond the age of 80 years.
African Sickle Cell News & World Report Oct-Dec 2016 Edition
These and more available for free at sickle cell news – hydroxyurea
New Sickle Cell Review Article
Good review article at http://www.medscape.com/viewarticle/872049: Management of Sickle Cell Disease in Children. Suzie A. Noronha, MD; S. Christy Sadreameli, MD, MHS; John J. Strouse, MD, PhD South Med J. 2016;109(9):495-502.
Articles in the medical literature
Pathology. 2016 Nov 30. pii: S0031-3025(16)40378-8. doi: 10.1016/j.pathol.2016.10.002. [Epub ahead of print]
Sickle cell disease in the older adult.
Thein MS1, Igbineweka NE2, Thein SL3.
Abstract
Sickle cell disease (SCD) is an inherited haemoglobin disorder, associated with recurrent painful episodes, ongoing haemolytic anaemia and progressive multi-organ damage. Until the early 1990s, survival beyond the fourth decade for a patient with SCD was considered unusual and prompted case reports. Nowadays, in countries with developed health care systems, more than 90 percent of newborns with SCD survive into adulthood. Nevertheless, their life expectancy is still shortened by more than two decades compared to the general population. With an increasing life expectancy, SCD has now evolved into a debilitating disorder with substantial morbidity resulting from ongoing sickle cell vasculopathy and multi-organ damage. Limited data on health care issues of older adults with SCD poses multiple challenges to patients, their families and health care providers. In this review, we will address and discuss acute and chronic complications of SCD with a special focus on the older adult.
Published by Elsevier B.V.
PMID: 27914684 [PubMed – as supplied by publisher]
Hematology Am Soc Hematol Educ Program. 2016 Dec 2;2016(1):640-647.
Sickle cell disease: an inherited thrombophilia.
Abstract
Activation of the hemostatic system occurs in patients with sickle cell disease. The extent to which this activation contributes to sickle cell pathophysiology is uncertain. Clinical trials of anticoagulants or platelet inhibitors have demonstrated the ability to decrease biomarkers of hemostatic activation, but this has generally not resulted in improvement in clinically relevant outcomes. Venous thromboembolism (VTE: deep venous thrombosis and pulmonary embolism) has been until recently an underappreciated complication of sickle cell disease, with incident event and recurrence rates consistent with a strong thrombophilia. There is no strong evidence that management should differ than for other patients with VTE, with the possible exception that secondary prophylaxis be extended regardless of provocation, given the persistent strong thrombophilic state.
© 2016 by The American Society of Hematology. All rights reserved.
PMID: 27913540 [PubMed – in process]
Hematology Am Soc Hematol Educ Program. 2016 Dec 2;2016(1):632-639.
Abstract
Since 1998, the National Institutes of Health has funded 5 randomized controlled trials (RCTs) for primary and secondary prevention of strokes in children with sickle cell anemia (SCA). In a systematic fashion, these trials have significantly advanced the care of children with SCA. In the absence of an RCT, clinicians are often compelled to make decisions at the bedside, based on experience, observational studies, and principles of hematology. We will provide an initial example that describes how a team-based, learning collaborative developed a multisite standard care protocol with a low budget (<$10 000 per year) to overcome the intrinsic limitations of advancing the care of neurologic complications in sickle cell disease (SCD). The critical components of this approach include: (1) regular meetings with the multidisciplinary team from multiple sites; (2) consensus regarding the best evidence-based neurologic management in multiple SCD centers; (3) an Institutional Review Board-approved protocol based on consensus standard care; (4) minimizing and ensuring accurate data collection; and most importantly, (5) a spirit of collaboration to improve the care of individuals with SCD. Four common neurologic problems and strategies for management in children and adults with SCD will be discussed: (1) secondary stroke prevention in high-income countries; (2) primary stroke prevention in low- and middle-income countries (LMICs); (3) poor academic performance in students; and (4) cognitive disability in adults. With a commitment to a team-based learning collaborative, incremental advances are possible for the neurologic care of children and adults with SCD.
© 2016 by The American Society of Hematology. All rights reserved.
PMID: 27913539 [PubMed – in process]
Hematology Am Soc Hematol Educ Program. 2016 Dec 2;2016(1):625-631.
Sickle cell disease: when and how to transfuse.
Howard J1.
Abstract
Blood transfusion remains an important therapeutic intervention in patients with sickle cell disease (SCD), aiming to both increase the oxygen carrying capacity of blood and to reduce the complications of vaso-occlusion. Simple, manual exchange and automated exchange can be effective in reducing the acute and chronic complications of SCD, and the advantages and disadvantages of each methodology mean they all have a role in different situations. Evidence for the role of emergency transfusion in the management of the acute complications of SCD, including acute pain and acute chest syndrome, comes from observational data. Several important randomized controlled trials have shown the efficacy of transfusion in primary and secondary stroke prevention in patients with SCD but, outside these areas, clinical practice lacks a clear evidence base. Evidence for the role of long-term transfusion in the prevention of the non-neurologic chronic complications of SCD comes from analysis of secondary outcomes of these randomized trials and from observational data. In view of the paucity of data, the risks and benefits of transfusion should be fully discussed with patients/families before a long-term transfusion program is commenced. Evidence is only available for the role of preoperative transfusion or for prophylactic transfusion through pregnancy in certain situations, and the role of transfusions outside these situations is discussed. Questions about when and how to transfuse in SCD remain and will need further randomized trials to provide answers.
© 2016 by The American Society of Hematology. All rights reserved.
PMID: 27913538 [PubMed – in process]
Haematologica. 2016 Dec 1. pii: haematol.2016.154245. [Epub ahead of print]
Associations between environmental factors and hospital admissions for sickle cell disease.
Piel FB1, Tewari S2, Brousse V3, Analitis A4, Font A5, Menzel S2, Chakravorty S2, Thein SL6, Inusa B7, Telfer P8, de Montalembert M3, Fuller GW5, Katsouyanni K4, Rees DC2.
Abstract
Sickle cell disease (SCD) is an increasing global health burden. This inherited disease is characterised by a remarkable phenotypic heterogeneity, which can only partly be explained by genetic factors. Environmental factors are likely to play an important role but studies of their impact on disease severity are limited and their results are often inconsistent. This study investigated associations between a range of environmental factors and hospital admissions of young patients with SCD in London and in Paris between 2008 and 2012. Specific analyses were conducted for sub-groups of patients with different genotypes and for the main reasons of admissions. Generalized additive models and distributed lag non-linear models were used to assess the magnitude of the associations and to calculate relative risks. Some environmental factors significantly influence the numbers of hospital admissions of children with SCD, although the associations identified are complicated. Our study suggests that meteorological factors are more likely to be associated with hospital admissions for SCD than air pollutants. It confirms previous reports of risks associated with wind speed (RR: 1.06/SD [95% confidence interval (CI): 1.00-1.12]) and also with rainfall (RR: 1.06/SD [95%CI: 1.01-1.12]). Maximum atmospheric pressure was found to be a protective factor (RR: 0.93/SD [95%CI: 0.88-0.99]). Weak or no associations were found with temperature. Divergent associations were identified for different genotypes or reasons of admissions, which could partly explain the lack of consistency in earlier studies. Advice to patients with SCD usually includes avoiding a range of environmental conditions that are believed to trigger acute complications, including extreme temperatures and high altitudes. Scientific evidence to support such advice is limited and sometimes confusing. This study shows that environmental factors do explain some of the variations in rates of admission to hospital with acute symptoms in SCD, but the associations are complex, and likely to be specific to different environments and the individual’s exposure to them. Furthermore, this study highlights the need for prospective studies with large numbers of patients and standardised protocols across Europe.
Copyright © 2016, Ferrata Storti Foundation.
Free Article
PMID: 27909222 [PubMed – as supplied by publisher]
Pediatr Blood Cancer. 2016 Dec 1. doi: 10.1002/pbc.26374. [Epub ahead of print]
Sobota AE1,2, Shah N3, Mack JW4,5.
Abstract
BACKGROUND:
Transition from pediatric to adult care is a vulnerable time for young adults with sickle cell disease (SCD); however, improvements in transition are limited by a lack of quality indicators. The purpose of this study was to establish quality indicators for transition in SCD and to determine the optimal timing between the final pediatric visit and the first adult provider visit.
PROCEDURE:
We conducted a modified Delphi survey to reach a consensus on which quality indicators are most important for a successful transition. Our expert panel consisted of members of the Sickle Cell Adult Provider Network. In the first round, the participants ranked a list of quality indicators by importance. In the second round, the participants chose their “top 5” quality indicators in terms of importance and also ranked them on feasibility.
RESULTS:
The response rates for the two rounds were 68 and 96%, respectively. Nine quality indicators were chosen as “top 5” by a majority of respondents, including communication between pediatric and adult providers, timing of first adult visit, patient self-efficacy, quality of life, and trust with their adult provider. Based on the comments from round 1, respondents were also asked for the optimal timing between leaving pediatric care and entering adult care. Most recommended a first adult visit within 2 months of the final pediatric visit.
CONCLUSIONS:
By using these quality indicators chosen by the majority of respondents, we can better develop and evaluate transition programs for young adults with SCD and improve health outcomes for these vulnerable patients.
© 2016 Wiley Periodicals, Inc.
PMID: 27905689 [PubMed – as supplied by publisher]
Clin Nephrol. 2016 Nov 30. [Epub ahead of print]
The kidney in sickle hemoglobinopathies .
Abstract
With improvements in the care of patients with sickle hemoglobinopathies, sickle cell disease (SCD) has evolved from a disease that was fatal in childhood into one in which most survive past their 5th decade and some into old age. As a result, the renal complications of sickle hemoglobinopathies, which are age dependent, have emerged as a common and serious complication of SCD. Approximately 14 – 18% of mortality in SCD is attributed to chronic kidney disease (CKD), which develops in 1/3 of individuals with SCD and progresses to end-stage renal disease in 4 – 18% of them. Importantly, the presence of CKD increases the risk of the other systemic complications of SCD, with the median survival of SCD estimated at 51 years, declining to 29 years in those with CKD. The obstructive vasculopathy of SCD affects the glomerulus, tubules, and medulla of the kidney. Albuminuria and inability to concentrate the urine precede the onset of renal failure, and, along with other tubular dysfunctions, are early warning signs of sickle cell nephropathy (SCN). This is a review of the historical background SCN, the pathophysiology of the renal lesions, their varied clinical and pathologic manifestations, and available treatment options. .
PMID: 27900941 [PubMed – as supplied by publisher]
Pediatr Blood Cancer. 2016 Nov 28. doi: 10.1002/pbc.26369. [Epub ahead of print]
Badawy SM1,2,3, Thompson AA1,2, Lai JS1,4, Penedo FJ4, Rychlik K1,5, Liem RI1,2.
Abstract
BACKGROUND:
Complications related to sickle cell disease (SCD) result in significant declines in health-related quality of life (HRQOL). While hydroxyurea reduces SCD complications, adherence remains suboptimal. The study’s objectives were to assess the feasibility of Internet-based electronic assessment of HRQOL in SCD clinic and to examine the relationship between HRQOL and hydroxyurea adherence in adolescents and young adults (AYAs) with SCD.
PROCEDURE:
A cross-sectional survey was administered on tablets to 34 AYAs (12-22 years old) in a SCD clinic from January through December 2015. Study measures included Patient Reported Outcomes Measurement Information System (PROMIS® ) computerized adaptive testing and ©Modified Morisky Adherence Scale 8-items (©MMAS-8).
RESULTS:
Participants (59% male, 91% Black) had median age of 13.5 (range 12-18) years. Ninety-one percent completed PROMIS® measures electronically in the clinic, meeting our feasibility criterion of ≥85% completion rate. ©MMAS-8 scores positively correlated with fetal hemoglobin (HbF) (rs = 0.34, P = 0.04) and mean corpuscular volume (MCV) (rs = 0.42, P = 0.01) and inversely correlated with fatigue (rs = -0.45, P = 0.01), depression (rs = -0.3, P = 0.08), and social isolation (rs = -0.78, P = 0.02). Low ©MMAS-8 scores, indicating poor adherence, were associated with worse fatigue (P = 0.001) and trended toward significance for pain (P = 0.07) and depression (P = 0.06). Homozygous hemoglobin S disease patients with low HbF (<16%) had worse social isolation (P = 0.04) and those with low MCV (<102 fl) reported worse fatigue (P = 0.001), pain (P = 0.01), mobility (P = 0.01), and social isolation (P = 0.04).
CONCLUSIONS:
HRQOL assessment in the SCD clinic is feasible. SCD patients with low hydroxyurea adherence and/or low HbF or MCV levels had worse HRQOL scores, particularly fatigue. Future prospective studies examining the relationship between HRQOL and hydroxyurea adherence are warranted.
© 2016 Wiley Periodicals, Inc.
PMID: 27896936 [PubMed – as supplied by publisher]
J Rare Dis Res Treat. 2016;1(2):25-31.
Gene therapy for hemoglobin disorders – a mini-review.
Abstract
Gene therapy by either gene insertion or editing is an exciting curative therapeutic option for monogenic hemoglobin disorders like sickle cell disease and β-thalassemia. The safety and efficacy of gene transfer techniques has markedly improved with the use of lentivirus vectors. The clinical translation of this technology has met with good success, although key limitations include number of engraftable transduced hematopoietic stem cells and adequate transgene expression that results in complete correction of β0 thalassemia major. This highlights the need to identify and address factors that might be contributing to the in-vivo survival of the transduced hematopoietic stem cells or find means to improve expression from current vectors. In this review, we briefly discuss the gene therapy strategies specific to hemoglobinopathies, the success of the preclinical models and the current status of gene therapy clinical trials.
TRIAL REGISTRATION:
ClinicalTrials.gov NCT01639690 NCT02186418 NCT02247843 NCT02453477.
PMCID: PMC5120727 Free PMC Article
PMID: 27891535 [PubMed]
Med Educ Online. 2016 Nov 24;21:33616. doi: 10.3402/meo.v21.33616. eCollection 2016.
Translating sickle cell guidelines into practice for primary care providers with Project ECHO.
Shook LM1, Farrell CB2, Kalinyak KA2, Nelson SC3, Hardesty BM4, Rampersad AG4, Saving KL5, Whitten-Shurney WJ6, Panepinto JA7, Ware RE2, Crosby LE8.
Abstract
BACKGROUND:
Approximately 100,000 persons with sickle cell disease (SCD) live in the United States, including 15,000 in the Midwest. Unfortunately, many patients experience poor health outcomes due to limited access to primary care providers (PCPs) who are prepared to deliver evidence-based SCD care. Sickle Treatment and Outcomes Research in the Midwest (STORM) is a regional network established to improve care and outcomes for individuals with SCD living in Indiana, Illinois, Michigan, Minnesota, Ohio, and Wisconsin.
METHODS:
STORM investigators hypothesized that Project ECHO® methodology could be replicated to create a low-cost, high-impact intervention to train PCPs in evidence-based care for pediatric and young adult patients with SCD in the Midwest, called STORM TeleECHO. This approach utilizes video technology for monthly telementoring clinics consisting of didactic and case-based presentations focused on the National Heart, Lung and Blood Institute (NHLBI) evidence-based guidelines for SCD.
RESULTS:
Network leads in each of the STORM states assisted with developing the curriculum and are recruiting providers for monthly clinics. To assess STORM TeleECHO feasibility and acceptability, monthly attendance and satisfaction data are collected. Changes in self-reported knowledge, comfort, and practice patterns will be compared with pre-participation, and 6 and 12 months after participation.
CONCLUSIONS:
STORM TeleECHO has the potential to increase implementation of the NHLBI evidence-based guidelines, especially increased use of hydroxyurea, resulting in improvements in the quality of care and outcomes for children and young adults with SCD. This model could be replicated in other pediatric chronic illness conditions to improve PCP knowledge and confidence in delivering evidence-based care.
Free Article
PMID: 27887664 [PubMed – in process]
Am J Hematol. 2016 Nov 23. doi: 10.1002/ajh.24612. [Epub ahead of print]
Daily pain in adults with sickle cell disease – a different perspective.
van Tuijn CF1, Sins JW1,2, Fijnvandraat K2, Biemond BJ1.
Abstract
Previous reports demonstrated that patients with sickle cell disease (SCD) experience pain on more than half of the observed days. Yet, these high incidences do not seem to match observations in our population. In this prospective cohort study we aimed to assess the frequency and characteristics of daily, self-reported pain among adult SCD patients in the Netherlands. Consecutive patients were enrolled during routine outpatient visits and followed up to 6 months. A total of 55 patients completed 5 982 diary observation days. Median age was 27 years (IQR 23-43). Patients reported SCD related pain on 17% of the observed days; on 13% of these days this pain was not defined as a painful crisis, while 3% was reported as a painful crisis but managed at home, and on 1% of the observed days patients were admitted to the hospital. Analgesics were used on 52% of days with pain with a relatively infrequent use of oral opioids (9% of pain days). This first European study on pain in SCD indicates that pain appears to be significantly less frequent in our population as compared to previous study cohorts from the United States, and may be more representative for current SCD populations in other Western countries. Besides a more widespread use of hydroxycarbamide in modern disease management, differences in organization and accessibility of healthcare between countries may also explain this discrepancy. This article is protected by copyright. All rights reserved.
© 2016 Wiley Periodicals, Inc.
PMID: 27880985 [PubMed – as supplied by publisher]
Blood. 2016 Nov 16. pii: blood-2016-07-727719. [Epub ahead of print]
Liem RI1, Chan C2, Vu TT2, Fornage M3, Thompson AA4, Liu K2, Carnethon MR2.
Abstract
The contribution of sickle cell trait (SCT) to racial disparities in cardiopulmonary fitness is not known despite concerns that SCT is associated with exertion-related sudden death. We evaluated the association of SCT status with cross-sectional and longitudinal changes in fitness and risk of hypertension, diabetes and metabolic syndrome over 25 years among 1,995 African Americans (56% women, 18 to 30 years old) in the Coronary Artery Risk Development in Young Adults (CARDIA) study. Overall, the prevalence of SCT was 6.8% (136/1,995) in CARDIA and over 25 years, 46% (738/1,590), 18% (288/1,631) and 40% (645/1,611) of all participants developed hypertension, diabetes and metabolic syndrome, respectively. Compared to participants without SCT, participants with SCT had similar baseline measures of fitness in cross-section, including exercise duration (535 vs. 540 sec, p = 0.62), estimated METs (11.6 vs. 11.7, p = 0.80), maximum heart rate (174 vs. 175 beats/min, p = 0.41) and heart rate at 2 minutes recovery (44 vs. 43 beats/min, p = 0.28). In our secondary analysis, there was neither an association of SCT status with longitudinal changes in fitness nor an association with development of hypertension, diabetes or metabolic syndrome after adjustment for sex, baseline age, BMI, fitness and physical activity. SCT is not associated with reduced fitness in this longitudinal study of young African American adults suggesting that the increased risk for exertion-related sudden death in SCT carriers is unlikely related to fitness. SCT status also is not an independent risk factor for developing hypertension, diabetes or metabolic syndrome.
Copyright © 2016 American Society of Hematology.
PMID: 27856464 [PubMed – as supplied by publisher]
Cochrane Database Syst Rev. 2016 Nov 14;11:CD007652.
Gene therapy for sickle cell disease.
Abstract
BACKGROUND:
Sickle cell disease encompasses a group of genetic disorders characterized by the presence of at least one hemoglobin S (Hb S) allele, and a second abnormal allele that could allow abnormal hemoglobin polymerisation leading to a symptomatic disorder.Autosomal recessive disorders (such as sickle cell disease) are good candidates for gene therapy because a normal phenotype can be restored in diseased cells with only a single normal copy of the mutant gene. This is an update of a previously published Cochrane Review.
OBJECTIVES:
The objectives of this review are:to determine whether gene therapy can improve survival and prevent symptoms and complications associated with sickle cell disease;to examine the risks of gene therapy against the potential long-term gain for people with sickle cell disease.
SEARCH METHODS:
We searched the Cochrane Cystic Fibrosis and Genetic Disorders Group Haemoglobinopathies Trials Register, which comprises of references identified from comprehensive electronic database searches and searching relevant journals and abstract books of conference proceedings.Date of the most recent search of the Group’s Haemoglobinopathies Trials Register: 15 August 2016.
SELECTION CRITERIA:
All randomised or quasi-randomised clinical trials (including any relevant phase 1, 2 or 3 trials) of gene therapy for all individuals with sickle cell disease, regardless of age or setting.
DATA COLLECTION AND ANALYSIS:
No trials of gene therapy for sickle cell disease were found.
MAIN RESULTS:
No trials of gene therapy for sickle cell disease were reported.
AUTHORS’ CONCLUSIONS:
No randomised or quasi-randomised clinical trials of gene therapy for sickle cell disease were reported. Thus, no objective conclusions or recommendations in practice can be made on gene therapy for sickle cell disease. This systematic review has identified the need for well-designed, randomised controlled trials to assess the benefits and risks of gene therapy for sickle cell disease.
PMID: 27841932 [PubMed – in process]
Cochrane Database Syst Rev. 2016 Nov 14;11:CD007175.
Antibiotics for treating osteomyelitis in people with sickle cell disease.
Martí-Carvajal AJ1, Agreda-Pérez LH.
Abstract
BACKGROUND:
Osteomyelitis (both acute and chronic) is one of the most common infectious complications in people with sickle cell disease. There is no standardized approach to antibiotic therapy and treatment is likely to vary from country to country. Thus, there is a need to identify the efficacy and safety of different antibiotic treatment approaches for people with sickle cell disease suffering from osteomyelitis. This is an update of a previously published Cochrane Review.
OBJECTIVES:
To determine whether an empirical antibiotic treatment approach (monotherapy or combination therapy) is effective and safe as compared to pathogen-directed antibiotic treatment and whether this effectiveness and safety is dependent on different treatment regimens, age or setting.
SEARCH METHODS:
We searched The Group’s Haemoglobinopathies Trials Register, which comprises references identified from comprehensive electronic database searches and handsearching of relevant journals and abstract books of conference proceedings. We also searched the LILACS database (1982 to 20 October 2016), African Index Medicus (20 October 2016), ISI Web of Knowledge (20 October 2016) and World Health Organization International Clinical Trials Registry Platform (20 October 2016).Date of most recent search of the Cochrane Cystic Fibrosis and Genetic Disorders Group’s Haemoglobinopathies Trials Register: 18 August 2016.
SELECTION CRITERIA:
We searched for published or unpublished randomised and quasi-randomised controlled trials.
DATA COLLECTION AND ANALYSIS:
Each author intended to independently extract data and assess trial quality by standard Cochrane methodologies, but no eligible randomised controlled trials were identified.
MAIN RESULTS:
This update was unable to find any randomised or quasi-randomised controlled trials on antibiotic treatment approaches for osteomyelitis in people with sickle cell disease.
AUTHORS’ CONCLUSIONS:
We were unable to identify any relevant trials on the efficacy and safety of the antibiotic treatment approaches for people with sickle cell disease suffering from osteomyelitis. Randomised controlled trials are needed to establish the optimum antibiotic treatment for this condition.
PMID: 27841931 [PubMed – in process]
Cochrane Database Syst Rev. 2016 Nov 14;11:CD005598.
Antibiotics for treating community-acquired pneumonia in people with sickle cell disease.
Martí-Carvajal AJ1, Conterno LO.
Abstract
BACKGROUND:
As a consequence of their condition, people with sickle cell disease are at high risk of developing an acute infection of the pulmonary parenchyma called community-acquired pneumonia. Many different bacteria can cause this infection and antibiotic treatment is generally needed to resolve it. There is no standardized approach to antibiotic therapy and treatment is likely to vary from country to country. Thus, there is a need to identify the efficacy and safety of different antibiotic treatment approaches for people with sickle cell disease suffering from community-acquired pneumonia. This is an update of a previously published Cochrane Review.
OBJECTIVES:
To determine the efficacy and safety of the antibiotic treatment approaches (monotherapy or combined) for people with sickle cell disease suffering from community-acquired pneumonia.
SEARCH METHODS:
We searched The Group’s Haemoglobinopathies Trials Register (01 September 2016), which comprises references identified from comprehensive electronic database searches and handsearching of relevant journals and abstract books of conference proceedings. We also searched LILACS (1982 to 01 September 2016), African Index Medicus (1982 to 20 October 2016) and WHO ICT Registry (20 October 2016).
SELECTION CRITERIA:
We searched for published or unpublished randomized controlled trials.
DATA COLLECTION AND ANALYSIS:
We intended to summarise data by standard Cochrane methodologies, but no eligible randomized controlled trials were identified.
MAIN RESULTS:
We were unable to find any randomized controlled trials on antibiotic treatment approaches for community-acquired pneumonia in people with sickle cell disease.
AUTHORS’ CONCLUSIONS:
The updated review was unable to identify randomized controlled trials on efficacy and safety of the antibiotic treatment approaches for people with sickle cell disease suffering from community-acquired pneumonia. Randomized controlled trials are needed to establish the optimum antibiotic treatment for this condition. The trials regarding this issue should be structured and reported according to the CONSORT statement for improving the quality of reporting of efficacy and improved reports of harms in clinical research. Triallists should consider including the following outcomes in new trials: number of days to become afebrile; mortality; onset of pain crisis or complications of sickle cell disease following community-acquired pneumonia; diagnosis; hospitalization (admission rate and length of hospital stay); respiratory failure rate; and number of participants receiving a blood transfusion.There are no trials included in the review and we have not identified any relevant trials up to September 2016. We therefore do not plan to update this review until new trials are published.
PMID: 27841444 [PubMed – in process]
Sickle Cell Conferences and Events
Foundation for Sickle Cell Disease Research Annual meeting
April 28 -30 2017 Ft Lauderdale FL http://fscdr.org/the-symposium/
