Sickle Cell Crisis Research Articles

Content validation of a self-report daily diary in patients with sickle cell disease

BackgroundSickle Cell Disease (SCD) is a genetic progressive vascular disease that impacts patients overall health and quality of life. Sickle-cell pain crises (SCPCs) are a hallmark clinical presentation of SCD and have been associated with increased morbidity and mortality. The Sickle Cell Pain Diary- Self Report (SCPD-S) was developed as a daily patient-reported outcome (PRO) measure primarily intended to capture the frequency and severity of SCD-related pain during and outside of a SCPC. The SCPD-S also examines the impact of the pain associated with an SCPC on other health-related quality of life concepts. The objective of this study was to investigate the content validity of the SCPD-S.MethodsThe content validation testing included 18 in-depth hybrid concept elicitation and cognitive debriefing interviews conducted with SCD patients in the US aged 12 years and older. Interviewers used a semi-structured interview guide and a think-aloud approach for the cognitive debriefing portion. All interviews were recorded, transcribed, coded and analyzed.ResultsEighteen interviews across two rounds were conducted. Round 1 hybrid interviews (n = 12) resulted in the expansion of the SCPD-S from 13 to 19 items. Items on the impact of an SCPC on social and recreational activities, sleep, and emotional well-being were added. Five items were significantly revised, as were three response choice sets. Round 2 hybrid interviews (n = 6) confirmed the comprehensiveness of the revised diary, understandability of the wording, and appropriateness of the recall period and response sets. Saturation analyses specific to concept elicitation revealed that no additional interviews were needed.ConclusionsThis study provided evidence to support the content validity of the SCPD-S, a self-report daily diary. Data gathered during patient interviews indicated that the SCPD-S is a fit for purpose measure of SCD and SCPC-related pain frequency and severity and the impact of this pain on other health-related quality of life concepts including fatigue and emotional health. The numerous changes to the SCPD-S as a result of the study findings highlight the importance of the content validation process when developing a PRO measure.

Dengue Fever Complicated by Sickle Cell Crisis with Multiple Splenic Infarcts

Dengue infections typically present with fever and thrombocytopenia. Although good number of patients improve with supportive care, few can have a fulminant course with multiorgan dysfunction. In endemic zone co-occurring illness like Sickle cell disease can contribute to poor outcome. Various splenic complications of Sickle cell disease include massive splenomegaly with sequestration, large infarcts and abscess requiring splenectomy. We report an interesting case of dengue fever, who developed shock and acute abdomen during hospital stay. Further evaluation revealed multiple splenic infarcts with correlating histopathology and etiological work up including hemoglobin electrophoresis helped in the de novo detection of the underlying sickle cell trait.International Journal of Human and Health Sciences Vol. 05 No. 04 October’21 Page: 519-524

Binasal Visual Field Defects Due to Sickle Cell Maculopathy.

A 26-year-old African American man with sickle cell disease noticed blurry vision in both eyes after a recent complicated hospital admission for sickle cell crisis. Anterior and posterior segment examination of each eye was normal, but visual field testing revealed binasal scotomas. Optical coherence tomography of the macula demonstrated severe thinning of the temporal inner retina, suggesting previous bilateral occlusions of terminal retinal arterioles involving the temporal macula, an uncommon complication of sickle cell disease. This case is a reminder that retinal pathology should always be considered as a potential cause of unexplained visual field defects and highlights the role of macular OCT in evaluation of these patients.

Serum Bone Menirals in Patients of Sickle Cell Anemia

Introduction: single nucleotide substitution A by T AT 17TH nucleotide of the 6th amino-acid Glutamic acid by valine in beta subunit of Hb (HbA) results in formation of sickle hemoglobin (HbS), Sickle bone syndrome is a public health problem to sicklers (disturbed serum bone minerals and bone density sickle cell crisis pain ). Because of genetic prevalence of sickle anaemia gene in Southern Sudan, sickle cell anaemia is a serious health issue and common cause of paediatrics admission at hospitals and the significance of measuring serum calcium, phosphorous and magnesium clarify the necessity to conduct this study. Which aimed to evaluate Ca, P and Mg Sudanese sicklers and correlate them to bone density and sickle cell crisis pain. Methods: A descriptive Case control study conducted in Southern Darfur Nyala-city involving Sudanese sicklers 50 and age matched control group 50,Hb electrophoresis records and clinical data for patients with sickle disease (known cases of SCD) serum Ca, P, Mg and Albumin were estimated using fully automated chemistry analyzer (Mindray BS-200) for All study groups. Data analysed using SPSS computer software v.24 Results: The results show normal magnesium levels with significant difference between serum magnesium levels among patients (2.43 (0.37) mg/dl) and controls (1.99 (0.47) mg/dl) p value 0.00, the mean serum calcium levels was significantly lower in the sickle cell disease group (8.4 (0.9 mg/dl) compared to controls (9.4 (0.6) mg/dl) p value 0.00. While the mean serum inorganic phosphorous was significantly higher in SCD group (6.6 (1.8) mg/dl) compared to control group (3.9 (0.82) mg/dl). Conclusion: Normomagnesimia hyperphosphatemia and hypocalcaemia are observed in SCD compared to controls.

Contrary to Expectations: Seasonal Variation in Sickle Cell Crisis — a Nation Wide Data Analysis

Background: Sickle cell crisis is a painful complication of sickle cell disease in adults and children. Acute episodes of severe pain (crises) are the primary reason for hospitalizations. Although infection, dehydration, and acidosis (all of which favor sickling) can act as triggers, in most instances, no predisposing cause is identified. Hospitalizations for sickle cell crisis have been steadily rising over the past decade. Though previous studies have described seasonal variations for in African and Caribbean populations, but there are no large data showing similar trends with patients in the United States. We reviewed a large national hospitalization database to determine whether rates of sickle cell crisis have seasonal variations over the past decade. Methods: We examined the Nationwide Inpatient Sample (NIS), a nationally representative survey of hospitalizations conducted by the Healthcare Cost and Utilization Project in collaboration with participating states. It is the largest all-payer inpatient dataset in the United States and includes a 20% sample of United States community hospitals that approximates 20% of all United States community hospitals. The Nationwide Inpatient Sample (NIS) database was used to estimate annual number of hospitalizations from 2000 - 2012. Identification of sickle cell disease related hospitalizations was based on the designation of the prior validated International Classification of Diseases (9th Edition) Clinical Modification (ICD-9-CM) diagnosis code 282.62 (Sickle cell crisis) as the principal discharge diagnosis. The frequency of hospitalization per month cumulative over 13 years was calculated and divided by number of days in that month to determine the mean hospitalizations per day for each month. All calculations were carried out using the weighted estimates approximating nationwide population estimates. Results: An estimated 744,072 hospitalizations with primary diagnosis of sickle cell crisis occurred in the US from 2000 to 2012. More specifically, the mean number of hospitalizations per day in each month is shown in Fig. 1. The mean number of hospitalizations each day was highest in January (2134 per day) and thereafter the hospitalization rate dropped to a nadir in June (1988 per day). There was a significant rising trend of admissions from June towards winter months with couple of peaks in September and January as depicted in the Fig. 1. In general, the number of hospitalization was maximum in the winter months and minimum in summer months as demonstrated in Fig .2. Conclusion: Contrary to expectations, we identified for the first time in United States an impressive pattern of seasonal variation in hospitalizations for sickle cell crisis with a notable increase in winter and fall and a significant drop during summer months. The seasonal pattern may reflect viral or other infections responsible for precipitation of sickle cell crisis. Otherwise, exposure to low temperatures causes increased viscosity of blood and peripheral vasoconstriction triggering sickle cell crisis. This statistical analysis calls for a definitive need to educate patients and providers on how to mitigate the adverse effects of weather on sickle cell crisis. Further efforts need to be done to identify triggers and methods to determine the relationship of these variations and reduce this predictive burden on the overall health care system. Download : Download high-res image (49KB) Download : Download full-size image Figure 1 . Disclosures No relevant conflicts of interest to declare.

Exploring Attitudes, Beliefs and Behavior Towards Hormonal Birth Control Among Female Patients with Sickle Cell Disease through Semi-Structured Interviews

▪Background: Sickle cell disease (SCD) is an inherited, autosomal recessive disorder and one way to decrease the risk of transmission of the disease is through informed reproductive choices. The U.S. Medial Eligibility Criteria for Contraceptive Use advises that the benefits of using hormonal contraceptives usually outweigh the risks for women with SCD. Recommendations for contraceptive use for individuals with sickle cell disease do not discourage the use of any specific contraceptives and identify sickle cell disease as a condition that exposes the woman to increased risk as a result of unintended pregnancy. Research indicates that women with SCD use hormonal contraceptives at lower rates than women without SCD.Objective: We sought to understand women with SCD knowledge, attitudes, and practices around hormonal contraceptives.Methods: African American females 18 and older with SCD were recruited from a sickle cell clinic in Atlanta, Georgia. We administered semi-structured interviews to examine the intersection of sickle cell and reproductive health. These semi-structured interviews explored participants' knowledge and attitudes towards hormonal contraceptives, as well as their personal experiences using hormonal contraceptives.Results: Seventeen women were interviewed on this topic. All but two of the participants indicated they had previously used hormonal contraceptives, but most of the women were not currently using hormonal birth control. Some of the participants had positive thoughts towards hormonal birth control because it prevents pregnancy.Birth control period. I am all for it. I feel like that is a good prevention of having unplanned pregnancies and whatever.Women cited positive experiences with hormonal birth control because it helped with heavy menstrual bleeding and decreased the number of sickle cell pain crises they experienced.I had depo before. It just made me gain weight and my cycles were off balance but it kind of helped with the Sickle Cell crisis a little bit.I would have pain crisis every time I had a menstrual cycle so my doctor told me to go to a gynecologist to get onto a birth control so that kind of decreased the amount of crisis that I was getting.Some of the participants had negative perceptions of hormonal birth control. Some were wary of it altogether. Most of them cited the side effects associated with hormonal birth control as the main reason they did not use them.I don't think any women should be on them just because of all the stuff in it. Some stuff they do not tell you what is in it and the weight gain and all the side effects I don't like them.That's why I don't like them because of the symptoms that I had. I wason Depo and I bleed for two months straight. And then I tried another one.Some green pill I don't know. I tried the implant. Like I said all the side effectsI didn't like them. I didn't like when I had side effects with it.Participants had specific concerns about the side effects related to menstrual bleeding and the intersection between contraceptive and sickle cell.I know they can just regulate your menstrual cycle. I kind of feel like that's unnatural. I think God put that in place for a reason and it needs to clean your body out. I just kind of think that part of it is unnatural and it shouldn't happen.I actually had to get one of them took out. The Nexplanon it actually caused a blood clot which is dangerous for a Sickle Cell patient.Right now I have an IUD and at first it was giving me back problems but I think it was just associated with Sickle Cell. So I think maybe it can trigger a Sickle Cell crisis sometimes.Conclusion: Our study findings indicate that women with SCD have low utilization of hormonal birth control with concerns for side effects caused by, and a general distrust of, hormonal contraception. However, some women also acknowledge that hormonal contraception can help prevent unplanned pregnancy, which not only may lead to unintended transmission of SCD, but also increased health risks to the woman. Providers caring for women with SCD may be able to better support them by adopting a patient-centered approach to contraceptive counseling. Seeking to clarify women's reproductive goals, contraceptive priorities and concerns, as well as correcting misinformation and reassuring women about hormonal contraceptive safety for women with SCD may be beneficial. DisclosuresNo relevant conflicts of interest to declare.

Safety of Deferiprone in Individuals with Sickle Cell Disease

Background: Deferiprone (Ferriprox®) is an oral iron chelator approved by the US Food and Drug Administration in 2011 for the treatment of transfusional iron overload in patients with thalassemia syndromes with inadequate chelation therapy. It has been used off-label for the treatment of transfusional iron overload in some patients with conditions other than thalassemia. Utilizing a comprehensive deferiprone patient registry, we sought to assess the safety profile of deferiprone use in patients with SCD.Methods: Since 2011, a patient registry has been maintained for all US patients who receive deferiprone, through a comprehensive support program, “Ferriprox Total Care” run by a central pharmacy. At registry entry, patient information is collected, including age, sex, and ICD-10 code for disease classification. The registry also collects all adverse effects (AEs) reported during deferiprone use through a detailed active drug surveillance program. Prior to initial drug shipment, patients are counseled by a pharmacist about the drug, invited to ask questions, and encouraged to contact the program with questions or safety concerns. Thereafter, the patient/family is contacted monthly to inquire about general well-being, which includes questions on dose adjustments, amount of drug left, hospitalizations, and side effects. Any patient reporting an AE is referred to a pharmacist, who collects additional information on the AE. All AEs are de-identified and reported to the Medical Safety department at ApoPharma. For each AE, the pharmacist and/or ApoPharma assigns causality to deferiprone, and the most conservative assessment prevails. This study reviewed all registry data through March 2016 to determine real-world use safety profile of deferiprone in patients with SCD.Results: A total of 291 patients with SCD, 43% male, received deferiprone. The mean age at drug initiation was 29.5 (SD 15.7) years (y) and 79 (27%) were younger than 18y. The mean drug exposure per patient was 1.3y (SD 1; range 0 to 4.1y). At the time of analysis, 117 (40%) remained on drug, 162 (56%) had discontinued treatment, and 12 (4%) were on hold in the registry for various reasons. 10% discontinued for AEs; AEs most commonly associated with discontinuation were nausea (48%), vomiting (31%), and sickle cell crisis (14%). Eighteen percent discontinued due to physician discretion and 10% due to switching to another chelator. Nine (3%) deaths were reported. All four deaths with follow-up information were deemed unrelated to Ferriprox use. Cause of death in the other five cases was not provided. Overall, a total of 275 AEs assessed as at least possibly related to deferiprone occurred in 112 patients (38.5%). These AEs are referred to as adverse drug reactions (ADRs). Gastrointestinal ADRs were most common including nausea (11%), vomiting (7%), and abdominal discomfort (6%). Arthralgia occurred in 6 patients (2%). Elevation in hepatic enzymes was noted in 6 patients (2.1%). In 61 patients (21%) 133 episodes of sickle cell crisis (36.44 events/100 pt years) were reported as serious AEs; five of these episodes were assessed as possibly related to deferiprone. Agranulocytosis (ANC <0.5 x109/L) developed in 2 (0.7%) patients (0.55 per 100 pt years) and milder neutropenia (ANC <1.5 x 109/L, but ≥ 0.5 x 109/L) in 3 (1%; 0.82 per 100 pt years).Conclusions: Deferiprone is frequently prescribed for patients with SCD. Agranulocytosis occurred in 0.7%, comparable to the rate of 1.7% seen in thalassemia. Overall, the adverse effect profile in patients with SCD is consistent with its known safety profile in thalassemia. DisclosuresKwiatkowski:Bluebird Bio: Research Funding; Apopharma: Research Funding; Novartis: Research Funding; Agios: Consultancy, Honoraria; Ionis: Consultancy, Honoraria. Tricta:ApoPharma: Employment. Rozova:ApoPharma: Employment.

Supplemental Oxygen during Air Travel in Sickle Cell Disease -Common Clinical Practices

Introduction: Air travel may expose patients with sickle cell disease (SCD) to an increased risk of disease related complications. Several factors are felt to contribute, including prolonged hypoxia, dehydration, temperature changes and stress. The Canadian Pediatric Society position statement recommends that SCD patients use supplemental oxygen on flights. While other notable guidelines including the National Heart, Lung and Blood Institute (NHLBI), the National Center for Biotechnology Information (NCBI), the Canadian Haemoglobinopathy Association (CanHaem) and the British Thoracic Society do not make specific recommendations. Since available guidelines and evidence lack consensus, it is unclear what recommendations healthcare practitioners (HCPs) should counsel to their SCD patients.Objectives:1.Determine the recommendations North American hematology HCPs make to SCD patients regarding air travel.2.Determine the proportion of HCPs recommending supplemental oxygen to SCD patients.3.Among HCPs that recommend supplemental oxygen, determine which patient factors influence their decision.4.Evaluate patient experiences regarding supplemental oxygen recommendations prior to air travel.Methods: First, a literature review using PubMed, TRIP database, Medline (Ovid) search engines was completed. Two investigators independently reviewed abstracts and references. Second, a cross-sectional online survey was circulated through CanHaem (186 members) and the American Society of Pediatric Hematology/ Oncology (ASPHO, 1991 members) listservs. North American hematology HCPs were asked to share their air travel recommendations. The survey was peer-reviewed by 7 practitioners prior to distribution for content and face validity. Similarly, a patient survey regarding experiences with air travel was distributed through the Sickle Cell Disease Association of Canada (SCDAC) listserv. University of Alberta ethics research board approved.Results: A review of the literature is summarized in Table 1. A total of 81 surveys were returned with 79 evaluable for analysis. Demographic data reveals that most survey respondents are physicians (90%), pediatric providers (77%), and practice at an altitude <100m above sea level (59%). Sixty-five percent practice in USA, and 35% in Canada. Eighty-two percent of HCPs do not recommend the use of supplemental oxygen for flights under any circumstance. Among those recommending oxygen, 9% recommend it for all patients, and 6% for HbSS/SB0 patients only. While most HCPs did not recommend oxygen, they did advise patients to increase hydration (94%), carry analgesics (94%), and wear warm clothes (83%). A few HCPs discouraged air travel altogether (12%). Air travel is considered a risk factor for vaso-occlusive crises (VOC) and acute chest syndrome (ACS) by 67% ad 37% of HCPs, respectively. Among HCPs recommending oxygen, most indicate that a previous history of VOC, stroke, silent infarct, ACS, splenic sequestration, or a history of altitude related complications increase the likelihood of recommending oxygen. Length of flight is an important consideration for HCPs recommending oxygen; 46% recommend oxygen for flights >6 hours, 31% for flights >10 hours and the remainder for all flight lengths. In a small sample of patients (n=5) the majority have experienced a sickle cell crisis during or shortly after air travel. Only one patient participant has used oxygen in the past.Conclusion: Most HCPs surveyed do not routinely recommend supplemental oxygen to their SCD patients as a prophylactic measure. Among HCPs that recommend oxygen both patient and flight factors influence their recommendations. Although oxygen is not a routine recommendation, most indicate that air travel is a likely risk factor for VOC. In fact, there are numerous published cases of air travel related complications, most commonly splenic complications and VOC, in the SCD population (Table 1). There is a lack of consensus among North American HCPs regarding supplemental oxygen recommendations. Further investigation is needed to delineate the need for supplemental oxygen to prevent complications during air travel for SCD patients. [Display omitted] DisclosuresBruce:Bristol-Meyers-Squibb - consultant 2016-2017: Consultancy; ovartis consult and ad board 2015-2016: Consultancy; Apopharma 2015: Consultancy.

Un vaccino falcemico

A case of a sickle cell crisis initially diagnosed as post vaccination arthritis is described. The author stresses the high specificity of acute dactylitis with respect to the diagnosis of sickle cell anaemia.

Non-Myeloablative Transplant in Severe Sickle Cell Disease Is Safe & Effective. Single Center Experience from Saudi Arabia

Background:Sickle cell disease (SCD) is a severe monogenic disorder in the world with >300,000 newly born afflicted children per year. Prevalence of this disorder is high in sub-Saharan Africa, Mediterranean basin, Middle East and India. Patients with severe disease phenotype manifested with stroke or other complications may benefit from allogeneic hematopoietic stem cell transplant (SCT). Recent reports in pediatric patients using myeloablative regimen showed excellent outcomes but similar progress in adults has been hampered by higher rates of transplant related mortality (TRM) and infertility.Methods:We conducted a study of adult patients at our institution with SCD whom underwent a matched related SCT during the period of 2014-2017. Medical records of eligible patients were retrospectively reviewed and extracted. All patients received hydroxyurea (HU) at maximally tolerated doses with hyper-transfusion 2-3 months prior to SCT. Iron chelation was given for patients with ferritin > 1000 ug/L. The preperative regimen was non-myeloablative (NMA) consisting of alemtuzumab 1 mg/kg divided over 5 days (day -7 to -3) followed by Total Body Irradiation (TBI) 300 cGy on day -1. Stem cells were collected from peripheral blood via apheresis after 5 days of GCSF with target CD34 cells of 10×106/kg. Graft vs. host disease (GVHD) prophylaxis consisted of sirolimus starting from day -2 and continued for one year post SCT with dose modification based on chimerism. Event free survival (EFS) was defined from time to SCT to graft failure, development of GVHD or death from any cause. EFS and overall survival (OS) via time to end point analysis was computed using the Kaplan-Meir test.Results:A total of 18 adults patients were eligible and further analysed. Median age was 23.5 (14 - 39) years with 14 males and 4 females. All patients were on HU prior to SCT. Median ECOG performance status was 1 (0-1). Indications for transplant were recurrent vaso-occlusive crisis (VOC) refractory to standard therapy in 61%, stroke or other central nervous system (CNS) complication in 38%, recurrent avascular necrosis of joints in 38%, recurrent acute chest syndrome in 22% and severe disease requiring recurrent intensive care unit admission (ICU) in 17%. All donors were related siblings fully matched at 10/10 human leucocyte antigen (HLA) loci with median age of 23.6 years. A total of 11 (61%) of donors were sickle cell carriers with average Hgb S 33%. Stem cell mobilization was with 5 ug/kg GCSF in all donors and the average stem cell collected 11.6 x 106/kg. No significant complications resulted from GCSF use in donors with sickle cell trait.17 patients were conditioned with alemtuzumab / TBI regimen and GVHD prophylaxis with sirolmus. One patient who failed prior transplant received fludarabine, busulfan and thymoglobulin with GVHD prophylaxis as cyclosporine and methotrexate. Median duration of neutrophil engraftment was 20 days and platelet engraftment 2.6 days with half of patient's platelet nadir > 20. Average hemoglobin (Hb) pre- and post-SCT was 83 g/L and 142 g/L respectively. Average hemoglobin (Hb) S pre- and post-SCT was 77% and 13% respectively. Peri-SCT adverse events were 9 (50%) patients experienced neutropenic fever, 12 (67%) mucositis, 6 (33%) diarrhea. None of the patients developed acute or chronic GVHD. No SCD crises developed post SCT. One patient developed aplastic anemia while retaining 71% donor cell chimerism and underwent a second successful SCT from the same donor with fludarabine, cyclophosphamide and thymoglobulin. All patients have stable mixed chimerism resulting in full donor myeloid engraftment and normalization of blood count. With a median follow up of 9.1 months (2.3-32), all patients are alive and free of sickle cell crises. At last follow up, OS and EFS were 100% and 88.9%, respectively as shown in Figure 1.Conclusion:Herein, we report that SCT in adults with severe SCD using NMA regimen is safe, feasible and results in amelioration of disease severity. These excellent results are encouraging given the high prevalence and debilitating nature of SCD in our region. Follow up is on-going to assess the feasibility of sirolimus tapering and sustainability of donor chimerism status over time. [Display omitted] DisclosuresNo relevant conflicts of interest to declare.

Sleep Patterns and Associated Clinical Correlates Amongst Ghanaian Individuals with Sickle Cell Disease

▪Background: Sickle cell disease (SCD) is the most commonly inherited blood disorder that affects 1 in 500 individuals of African descent worldwide and 70,000-80,000 individuals in the United States. While the molecular basis and progression of this condition has been well established, the association between sleep, stress and frequency of complications such as pain crises has not been well studied. In this study, we investigate how sleep behavioral patterns are associated with the severity and incidence of sickle cell crises amongst patients with SCD.Methods: Adult individuals with SCD were recruited from the Korle Bu Teaching Hospital in Accra, Ghana as participants for this study. No compensation was provided. Participants completed a questionnaire that assessed SCD disease severity such as frequency of pain crises and need for hospitalization. Self-reported sleep habits, including average hours of sleep both at baseline and during pain crisis were also evaluated. Medical records for each participant were also obtained to establish demographic and genotype information.Results: A total of 308 individuals with SCD were enrolled in the study. Patients were classified according to SCD genotype with 68.6% of individuals with severe genotype (hemoglobin SS or sickle/beta thalassemia), and 31% had mild genotype (hemoglobin SC, sickle/beta plus thalassemia disease). Regarding average sleep per night, 2.4% reported 0-2 hours, 14.4% reported 3-4 hours, 38.8% reported 5-6 hours, 36.8 reported 7-9 hours, and 7.7% reported 10 hours or more. During pain crises, 48% reported 0-2 hours of sleep and 91.1% reported 0-4 hours of sleep per night. A paired sample t-test of average hours of sleep at baseline versus sleep during pain crisis showed a significant decrease in hours of sleep while in pain crisis (t=20.16, p < 0.001). Furthermore, female gender was associated with decreased sleep during pain crises (p = 0.008) compared to their male counterparts. An analysis of all SCD Genotypes revealed average sleep per night at baseline was not associated with frequency of crises per year, pain crises requiring ER/doctor visits nor per pain crises per year requiring hospitalizations. However, within the mild genotype group alone, average hours of sleep during a pain crisis was significantly associated with number of crises per year (r = 0.299, p = 0.04), number of crises/year requiring ER/doctor visit (r = 0.256, p = 0.013), and crises/year requiring hospitalization per year (r = 0.246, p = 0.018). Sleep patterns and pain crises/year measures did not correlate amongst patients with severe genotype.Discussion: Per patient self-report, individuals with SCD experience significant disruption with sleep due to pain crises. Most of the individuals report a significant reduction in average amount of sleep during a pain crisis, suggesting that pain crisis likely plays a crucial role in quality of life amongst patients with SCD. In patient with milder genotypes, worse outcomes (frequent ER/doctor visits, frequent crises, and frequent hospitalizations) are associated with less sleep during a pain crisis. This association suggests that amongst those with mild genotypes, sleep amount likely reflects SCD morbidity. The lack of associated pain crises patterns and sleep within the severe genotype suggests most adult patient regardless of severity report a severe lack of sleep during painful crises. Leg ulcer associated with less sleep during painful crises may reflect the impact of leg ulcers on sleep patterns. A limitation of this study was that data was obtained from a single site which may not reflective of sleep patterns amongst individuals with SCD elsewhere. Future areas of exploration could include evaluation of sleep habits with a validated sleep questionnaire and compared with end organ complications of sickle cell disease. DisclosuresWilson:American Society of Hematology: Research Funding. Asare:Office of Research Innovation and Development (ORID) Research Fund/8/LMG-008, University of Ghana; Doris Duke Charitable Foundation; Burroughs Wellcome Foundation; Phillips Family Donation; Aaron Ardoin Foundation for Sickle Cell Anemia; Vanderbilt Univer: Research Funding. Olayemi:Office of Research Innovation and Development (ORID) Research Fund/8/LMG-008, University of Ghana; Doris Duke Charitable Foundation; Burroughs Wellcome Foundation; Phillips Family Donation; Aaron Ardoin Foundation for Sickle Cell Anemia; Vanderbilt Univer: Research Funding.

Un vaccino falcemico

A case of a sickle cell crisis initially diagnosed as post vaccination arthritis is described. The author stresses the high specificity of acute dactylitis with respect to the diagnosis of sickle cell anaemia.

Relationship between disease severity and folate status of children with sickle cell anaemia in Enugu, South East Nigeria.

Repeated crises in children with sickle cell anaemia (SCA), which is a manifestation of disease severity, results in depletion of their minimal tissue folate stores, with higher likelihood of folate deficiency. The study aimed to determine the relationship between disease severity and the folate status of children with SCA attending University of Nigeria Teaching Hospital (UNTH), Enugu. This was a hospital based, cross-sectional study conducted between September 2018 and March 2019. One hundred participants were recruited, consisting of 50 children having sickle cell crisis and 50 age and gender matched haemoglobin AA genotype controls. Relevant information was documented using a pretested questionnaire. Sickle cell severity score was determined using frequency of crisis, admissions and transfusions in the preceding one year, degree of liver and splenic enlargement, life-time cummulative frequency of specific complications of SCA, leucocyte count and haematocrit. Folate deficiency was observed in eight percent of the subjects and none of the controls. The difference was not significant (Fisher's exact = 4.167, p=0.117). The odds of being folate deficient was 8.5 times more likely during anaemic crisis than in vaso-occlusive crisis, though not significant (95% C.I 0.05 - 89.750, p = 0.075). The mean SCA severity score was 8.06 ± 3.64, signifying a moderate SCA severity in the study population. There was a no relationship between folate status and severity of SCA (Fisher's exact = 0.054, p = 0.949). Folate status in children with SCA is not affected by their disease severity. Therefore, there may be no need for additional folate supplementation with increasing severity of sickle cell anaemia.

Cardiovascular benefits of a home-based exercise program in patients with sickle cell disease.

BackgroundPhysical inactivity is an important risk factor for cardiovascular disease. The benefits of exercise in patients with chronic diseases, including cardiovascular diseases, are well established. For patients with sickle cell disease, medical recommendation was to avoid physical exercise for fear of triggering painful crises or increasing the impairment of the cardiopulmonary function. Only recently, studies have shown safety in exercise programs for this population. Despite that, there is no report that assess the effects of physical exercise on cardiac parameters in patients with sickle cell disease.ObjectiveThis study aimed to evaluate the impact of regular physical exercise (a home-based program) on cardiovascular function in patients with sickle cell disease.DesignA quasi-randomized prospective controlled trial.SettingDuring the years 2015 and 2016, we started recruiting among adult patients treated at a Brazilian Center for Patients with Sickle Cell Disease to participate in a study involving a home exercise program. The experimental (exercise) and control groups were submitted to clinical evaluation and cardiovascular tests before and after the intervention. Analysis of variance was applied to compare groups, considering time and group factors.ParticipantsTwenty-seven adult outpatients with a sickle cell disease diagnosis.InterventionsExercise group (N = 14): a regular home-based aerobic exercise program, three to five times per week not exceeding give times per week, for eight weeks; no prescription for the control group (N = 13).Main outcome measuresEchocardiographic and treadmill test parameters.ResultsThe exercise group showed significant improvement in cardiovascular tests, demonstrated by increased distance traveled on a treadmill (p<0.01), increased ejection fraction (p < 0.01) and improvement of diastolic function assessed by mitral tissue Doppler E’ wave on echocardiography (p = 0.04). None of the patients presented a sickle cell crisis or worsening of symptoms during the exercise program.ConclusionThe selected home-based exercise program is safe, feasible, and promotes a favorable impact on functional capacity and cardiovascular function in sickle cell disease patients.

551 Outcomes of Sickle Cell Crisis in Relation to Pediatric Obstructive Sleep Apnea in the United States: A US Population cohort study

Abstract Introduction This study aimed to quantify the impact of obstructive sleep apnea (OSA) on the mortality, morbidity, and resources utilization among children admitted with Sickle cell crisis (SCC) Methods This is a retrospective analysis using the 2016 and 2017 National Inpatient Sample Database. The Inclusion Criteria was a principal Diagnosis of SSC and age &amp;lt;18 years. OSA, as a Secondary Diagnosis, was identified using the appropriate ICD-10 CM codes. The Primary Outcomes were Inpatient Mortality, and secondary outcomes were: In-Hospital Length of Stay(LOS), Total hospitalization Cost, Blood transfusion (BT) requirement, and a requirement for Invasive mechanical ventilation (IMV). We used Multivariate Linear/ logistic regression to adjust for confounders including age and sex. Results Out of 36,484 children with SSC included in the study, 1450 children had OSA (SCC+OSA). SSC-OSA and SSC+OSA groups did not differ in gender, household income, and hospital characteristics, but did so in age (11.3 vs 12.4; p &amp;lt;0.001). OSA was most common in the age group of 13–18 (54%) and lowest in 0–4 (2.4%). Compared to SSC-OSA, the SCC+OSA cohort had significantly higher odds of mortality (adjusted OR= 11.9, [95% Confidence Interval: 1.02- 138.8],p=0.04). Additionally, SSC+OSA cohort was associated with increased odds of IMV (aOR=5.24 [CI: 1.84 – 14.8], p=0.002), longer LOS (adjusted mean difference (aMD)=0.67 [CI-0.32 – 1.02], p=&amp;lt;0.001), and higher hospitalization Cost (aMD=2818.76 [CI-1680- 4157], p=&amp;lt;0.001). No difference in BT (aOR=0.94 [CI: 0.68 – 1.29], p=0.71) was noted. Conclusion This study demonstrates that the presence of OSA is associated with detrimental outcomes in SSC with higher in-hospital mortality, higher morbidity (Invasive mechanical ventilation rate), and higher resource utilization (LOS, total hospitalization cost). More attention to the screening, early diagnosis, and appropriate treatment of OSA is imperative to improve health outcomes in children with sickle cell disease. Support (if any):

Association between Sickle Cell Crisis and Other Types of Hemoglobin among Sudanese Children with Sickle Cell Anemia

Association between Sickle Cell Crisis and Other Types of Hemoglobin among Sudanese Children with Sickle Cell Anemia

Osteonecrosis of mandible secondary to sickle cell crisis

<h3>Background</h3> Sickle cell disease (SCD) is an autosomal recessive hemoglobinopathy causing amino acid substitution in the B-hemoglobin chain of erythrocytes. SCD affects 1 in 500 African American children. SCD is associated with multiorgan morbidity and significant reduction in life expectancy. Vaso-occlusive crisis associated with SCD is characterized by acute pain and local tissue hypoxia and necrosis. The extremities, back, and chest are frequently affected, and jaw involvement is very rare. To the best of our knowledge, only 3 cases of jaw osteonecrosis secondary to SCD have been reported in the English-language literature. <h3>Case Summary</h3> A 38-year-old African American man with a history of SCD and avascular necrosis of the hips presented to an emergency department with severe pain in his back, legs, and chest due to a vaso-occlusive episode precipitated by physical overexertion. A few days later, the patient complained of left jaw pain and paresthesia. Computed tomography (CT) of the head with intravenous (IV) contrast, maxillofacial CT with IV contrast, and the remainder of the neurologic examinations were unremarkable. The oral medicine service was consulted, and physical examination of the patient demonstrated lingual gingival tissue swelling of the lower left molars. The mass was nonfluctuant with ulceration causing malalignment of tooth number 18. Bone biopsy revealed squamous mucosa with hemorrhagic foci, ulceration, and ectatic blood vessels with fragments of necrotic bone consistent with osteonecrosis. At the patient's 2-week follow-up visit, a small area of exposed necrotic bone was noted in the affected area. Maxillofacial CT without IV contrast showed mixed radiolucency and sclerosis with dehiscence along the lingual cortex of the posterior mandibular body and along the cortex of the retromolar trigone. Chlorhexidine gluconate 0.12% oral solution was prescribed, and debridement of the left posterior mandible and extraction of the lower left molars with local flap reconstruction were recommended by an oral and maxillofacial surgeon. The patient did not return for follow-up management. <h3>Conclusions</h3> Acute pain and chronic vasculopathy are significant complications of SCD. Jaw involvement in SCD is very rare, as illustrated in this case. It is important for oral health care professionals to understand the pathophysiology and clinical manifestations of SCD.

Young child with painful edema and purpura: a case report

BackgroundWe reported the case of a two-old-year boy with a painful acute hemorrhagic edema. This is a self-limited benign condition: usually, affected children are well appearing and this strongly support the diagnosis. In the opposite, in our case, we observed a painful presentation of the edema. Therefore, we demonstrated that rarely, this condition could have also a painful presentation.ConclusionsThis case report helps clinician to know that also acute hemorrhagic edema could have a painful presentation, so we must considered it in the differential diagnosis with sepsis, sickle cell crisis and child abuse. We believe that these findings will be of interest to pediatricians.

Agonistic Anti-CD40 Antibody Triggers an Acute Liver Crisis With Systemic Inflammation in Humanized Sickle Cell Disease Mice.

Sickle cell disease (SCD) is an inherited hemolytic disorder, defined by a point mutation in the β-globin gene. Stress conditions such as infection, inflammation, dehydration, and hypoxia trigger erythrocyte sickling. Sickled red blood cells (RBCs) hemolyze more rapidly, show impaired deformability, and increased adhesive properties to the endothelium. In a proinflammatory, pro-coagulative environment with preexisting endothelial dysfunction, sickled RBCs promote vascular occlusion. Hepatobiliary involvement related to the sickling process, such as an acute sickle hepatic crisis, is observed in about 10% of acute sickle cell crisis incidents. In mice, ligation of CD40 with an agonistic antibody leads to a macrophage activation in the liver, triggering a sequence of systemic inflammation, endothelial cell activation, thrombosis, and focal ischemia. We found that anti-CD40 antibody injection in sickle cell mice induces a systemic inflammatory and hemodynamic response with accelerated hemolysis, extensive vaso-occlusion, and large ischemic infarctions in the liver mimicking an acute hepatic crisis. Administration of the tumor necrosis factor-α (TNF-α) blocker, etanercept, and the heme scavenger protein, hemopexin attenuated end-organ damage. These data collectively suggest that anti-CD40 administration offers a novel acute liver crisis model in humanized sickle mice, allowing for evaluation of therapeutic proof-of-concept.

Lived Experience of Sickle Cell Patients during and after Crisis

Aims and Objectives: To understand the lived experience and needs of patients with sickle cell disease during and two weeks after their crisis and identify the obstacles faced by patients while they are in the hospital. Background: Although there is no specific data of a number of affected individuals with sickle cell disease in Oman based on their age, the majority of the Omani population are youth. This category of the population is either in their high school or working in the governmental or private sector in the country. When the most productive category of the population are getting frequently absent due to sickle cell crisis and complication of sickle cell crisis from their work, this leads to huge financial and human resource burden. Design: Phenomenology. Method: This qualitative descriptive research was conducted using face-to-face interviews based on an interview protocol. The interview protocol was developed by the authors based on a framework called domains of well-being. Twenty adult patients have been recruited for the interview after meeting inclusion criteria and were asked about their well-being and lived experience during sickle cell crisis. Authors used SRQR checklist in reporting the study. Results: Thirteen themes were identified related to patients’ lived experience and their well-being during sickle cell crisis. Patients reported physical, emotional, social, and spiritual alteration. Major themes that emerged are communication, medical team interpretation of genuine pain, Emotional disturbance during the crisis, What does this study contribute to the wider global clinical community? Nurses and doctors should use therapeutic communication when dealing with sickle cell patients. Nurses should establish rapport and trust with patients. In each health care setting, there should be a social worker to deal with patients with chronic illness social relationships between the patient, family and friends, post-discharge status, spiritual and Islamic activities, and physical abilities. Conclusion: Participants’ physical and psychological statuses were mostly affected. Moreover, participants experienced extreme emotional disturbance during a painful crisis. However, it was not well understood why participants experienced post sickle cell crisis symptoms which need to be further investigated. Relevance to Clinical Practice: Understanding the lived experience of sickle cell patients may help improve nursing and medical care provided to them and enhance better outcomes for patients. These findings made the nurses and physicians plan a strategy of treating sickle cell patients using a holistic approach.