Survival of total hip arthroplasty (THA) in sickle cell disease.

BACKGROUND: The objective of this study was to evaluate the outcome of THA done in patients with sickle cell disease at a single center, and compare with rest of the world before we could recommend routine THA in SCD patients. METHODS: In 92 patients, 117 THAs were performed in sickle cell patients. Seventy-six (76) patients had homozygous hemoglobin (Hgb SS) disease, whereas sixteen had sickle cell trait (Hgb AS). Patients with Grade III Ficat and above avascular necrosis of the head of femur (ANFH) with cementless porous-coated proximal femur fixation were included in this analysis. Harris hip score was modified (MHHS) and used for assessment of improvement and visual analog scale score (VAS), for pain assessment. RESULT: VAS improved from average pre-operative mean of 7 (7-9) to 2.5 (1-5). The average pre-operative MHHS was 44 (32-63), and it improved to 79 (71-88) in the final mean follow-up of 12.4 (4-18) years. Nine patients (9.7%) had complications and revision surgery was required in seven (5.9%) for aseptic loosening. CONCLUSION: Our analysis shows that THA in sickle cell disease is safe with average 12 years of follow-up, indicating that surgery in these young patients (≤ 25 years) is safe and should not be delayed.

Femoral Head Core Decompression and Bone Marrow Concentrate Injection in Pediatric Sickle-cell Related Avascular Necrosis.

BACKGROUND: Femoral head avascular necrosis affects 10% to 40% of patients with sickle cell disease (SCD). The purpose of this study was to evaluate the results of femoral head core decompression and bone marrow aspirate concentrate (CD-BMAC) injection in pediatric patients with SCD. METHODS: Eligible patients were enrolled in this retrospective study of patients undergoing CD-BMAC. Patients with SCD who were younger than 18 at the time of surgery and had >1 year of follow-up were included in this analysis. Hips were staged based on the Ficat system by 2 raters. The visual analog score for pain, hip outcome score, modified Harris hip score, and the University of California, Los Angeles activity score were used as patient-reported outcome measures preoperatively, at 5 to 9 months postoperatively, and final follow-up. Treatment failure was defined as total hip arthroplasty or visual analog score >3 at the final follow-up. RESULTS: Twenty-three hips in 17 patients were included, with a median age at the time of surgery of 15.8 years (interquartile range: 13.1 to 17.8 y). Patients were followed for 4.25 ± 1.7 years. Ten hips showed a 1-stage increase in the Ficat stage at the final follow-up, whereas 11 retained the same stage, and 1 hip had a 1-stage regression. All patient-reported outcome measures showed significant improvement from the preoperative visit to short-term follow-up, but the preoperative to final follow-up improvement was not significant. Six treatment failures were recorded, including 3 total hip arthroplasties and 3 painful hips at the final follow-up. In a multivariate logistic regression model, only skeletal maturity (odds ratio = 16.2, 95% CI: 1.44-183.0, P = 0.024) and femoral head collapse (odds ratio = 12.0, 95% CI: 1.1-130.5, P = 0.041) were significant predictors of treatment failure. CONCLUSIONS: In the largest study on pediatric SCD patients undergoing CD-BMAC, we found that CD-BMAC injection offers significant improvement in pain and functional outcomes in the short term, with a very low risk for complications. Skeletal maturity and femoral head collapse were significant predictors of treatment failure. Patients with a collapsed femoral head experienced functional deterioration over time after initial improvement, whereas precollapse hips maintained their functional improvement up to the latest follow-up. LEVEL OF EVIDENCE: Level III, retrospective cohort study.

How does sickle cell disease affect the peri-operative outcome in patients undergoing total knee arthroplasty? A large-scale, National Inpatient Sample-based study.

INTRODUCTION: In view of the vaso-occlusive pathophysiology affecting osseous micro-circulation, sickle cell disease (SCD) is well known to present with diverse skeletal and arthritic manifestations. With prolonged life-expectancy over the past decades, there has been a progressive increase in the proportion of SCD patients requiring joint reconstructions. Owing to the paucity of evidence in the literature, the post-operative complication rates and outcome in these patients following total knee arthroplasty (TKA) are still largely unknown. METHODS: Based on the National Inpatient Sample (NIS) database (using ICD-10 CMP code), patients who underwent TKA between 2016 and 2019 were identified. The cohort were classified into two groups: A-those with SCD; and B-those without. The data on patients' demographics, co-morbidities, details regarding hospital stay including expenditure incurred, and complications were analyzed and compared. RESULTS: Overall, 558,361 patients underwent unilateral, primary TKA; among whom, 493 (0.1%) were known cases of SCD (group A). Group A included a significantly greater proportion of younger (60.14 ± 10.87 vs 66.72 ± 9.50 years; p < 0.001), male (77.3 vs 61.5%; p < 0.001); and African-American (88.2 vs 8.3%B; p < 0.001) patients, in comparison with group B. Group A patients were also at a significantly higher risk for longer duration of peri-operative hospital stay (p < 0.001), greater health-care costs incurred (p < 0.001), and greater need for alternative step-down health-care facilities (p < 0.001) following discharge. Among the SCD patients, 24.7%, 20.9% and 24.9% developed acute chest syndrome, pain crisis and splenic sequestration crisis, respectively during the peri-operative period. Group A patients had a statistically greater incidence of acute renal failure (ARF; p = 0.014), need for blood transfusion (p < 0.001) and deep vein thrombosis (DVT; p = 0.03) during the early admission period. CONCLUSION: The presence of SCD substantially lengthens the duration of hospital stay and enhances health care-associated expenditure in patients undergoing TKA. SCD patients are at significantly higher risk for systemic complications including acute chest syndrome, pain crisis, splenic sequestration crisis, acute renal failure, higher need for blood transfusions and deep venous thrombosis during the initial peri-operative period following TKA.

Impact of sickle cell disease on postoperative outcomes following total hip arthroplasty.

INTRODUCTION: Osteonecrosis (ON) of the femoral head is a common complication of sickle cell disease (SCD), frequently necessitating total hip arthroplasty (THA). Although THA is a common procedure with few complications when indicated for osteoarthritis (OA), patients with SCD have increased rates of complications. The purpose of this study is to determine the impact of SCD on outcomes and complications following THA. METHODS: Subjects were retrospectively identified using a national insurance claims database (PearlDiver Technologies) using CPT and ICD-9/10. Patients were included if they underwent THA with at least 2-year follow-up and were diagnosed with SCD and ON, ON without SCD, or OA without SCD. The SCD cohort was matched based on age, gender, Charlson Comorbidity Index, and obesity to the other 2 cohorts. 2-year revision rate and 90-day complication rates were analyzed using chi-square tests. RESULTS: Each cohort had 881 patients. The SCD cohort had significantly higher 90-day medical complications when compared to the ON without SCD and OA cohorts (p < 0.001). The SCD cohort had a higher rate of 2-year revision for PJI (5.0%) compared to the ON without SCD (2.8%) and OA (2.5%) groups (p = 0.019 and 0.005 respectively) and a higher rate of aseptic loosening (1.94%) compared to the ON without SCD cohort (0.68%; p = 0.021). CONCLUSIONS: Patients with SCD who undergo THA have an increased risk for short-term postoperative medical complications and 2-year aseptic loosening and PJI compared to ON patients without SCD and patients with OA. Given the magnitude of these risks, patient counseling and optimised perioperative care are essential.

Outcomes of Patients With Sickle Cell Disease and Trait After Congenital Heart Disease Surgery.

BACKGROUND: Sickle cell disease (SCD) is a hemoglobinopathy that can cause multiorgan dysfunction. This study assessed the perioperative outcomes of patients undergoing operations for congenital heart disease who had SCD or sickle cell trait (SCT). METHODS: We performed a retrospective review of patients with SCD or SCT who had records in The Society of Thoracic Surgeons Congenital Heart Surgery Database between 2014 and 2019. The primary outcome was operative mortality. Secondary outcomes included postoperative complications. One-to-one propensity score matching was performed between the SCD and SCT groups and the control group for further analysis. RESULTS: Our study population consisted of 73, 411, and 36 501 patients in the SCD, SCT, and control groups, respectively. Median (25%-75% interquartile range) age at surgery was 2.8 (0.4-9.7), 0.60 (0.2-3.1), and 0.70 (0.2-6.4) years in the SCD, SCT, and control cohorts, respectively. Operative mortality, surgery duration, cardiopulmonary bypass time, and cross-clamp time were not significantly different among the 3 groups. The SCD group had a higher rate of postsurgical cardiac arrest than its propensity score-matched control group (5.5% vs 0%, P < .05); otherwise, there were no statistically significant differences in the outcomes between the SCD and SCT groups and their respective matched control groups. CONCLUSIONS: Operative mortality after cardiothoracic procedures in patients with SCD or SCT appeared similar to our control patients. While these patients may require unique perioperative management, they can undergo cardiac surgery without an observed increase in mortality.

Modifying priming techniques in cardiopulmonary bypass circuit in known case of sickle-cell trait undergoing open heart surgery.

Modifying cardiopulmonary bypass (CPB) circuit's priming technique before the onset of CPB in cardiac patients known with sickle-cell disease or sickle cell trait has been observed to be of substantial significance in dealing with such challenges without having any life-threatening consequences. We modified our routine heparinized crystalloid priming of the CPB circuit with partial exchange transfusion by adding donor blood (packed red blood cells), fresh frozen plasma (FFP), and bicarbonate. This has helped us bring down the overall sickle cell hemoglobin in the blood thereby reducing its risk of sickling.

An Overview of Solid Organ Transplantation in Patients With Sickle Cell Disease.

Sickle cell disease is a common genetic disorder affecting >300 000 people across the world. The vast majority of patients cared for in high-resource settings live well into adulthood, but many develop a high burden of disease complications. Good standard of care including disease-modifying agents and transfusion programs limits the number of patients who develop end-stage organ disease, but for those that do, the prognosis can be very poor. Solid organ transplantation is a well-established mode of treatment for patients with sickle cell disease and kidney or liver failure, but appropriate patient selection and perioperative management are important for achieving good outcomes. Hematopoietic stem cell transplantation and gene therapy may offer novel treatment options for adult patients with chronic organ damage in the future, but these are not yet widely available. For now, good, holistic care and early intervention of end-organ complications can minimize the number of patients requiring solid organ transplantation later in life.

Impact of sickle cell disease in shoulder arthroplasty: a matched cohort analysis.

BACKGROUND: Sickle cell disease (SCD) is a genetic disorder of abnormal hemoglobin synthesis that is known to cause glenohumeral avascular necrosis (AVN). Little has been published on the use of shoulder arthroplasty (SA) for the treatment of glenohumeral AVN in SCD. We report on the clinical and radiographic results and postoperative complications following SA in the patient cohort. METHODS: A retrospective review was performed identifying 17 primary SAs (9 hemiarthroplasties, 7 anatomic total SAs, and 1 reverse total SA) in patients with a confirmed diagnosis of SCD and a minimum of 2-year follow-up. This cohort was matched (1:2) according to age, sex, body mass index, type of prosthesis, and year of surgery with patients who had undergone hemiarthroplasty or total SA for osteoarthritis (OA) or reverse total SA for cuff tear arthropathy. Outcomes included the visual analog scale (VAS) for pain, American Shoulder and Elbow Surgeons score, range of motion, and strength measurements in forward elevation (FE), external rotation (ER), and internal rotation (IR). RESULTS: Our cohort included 9 (52.9%) men with a mean age of 43 yr. The average follow-up time was 5.9 yr. In patients with SCD, SA provided significant improvements in VAS pain (9.1-3.8; P < .001), FE (95°-128°; P < .001), ER (24°-38°; P < .001), IR score (3.2-5.2; P < .001), FE strength (4.2-4.8; P < .001), ER strength (4.1-4.7; P < .001), IR strength (4.1-4.7; P < .001), and American Shoulder and Elbow Surgeons scores (48.6-73.5; P < .001). When compared to the matched cohort, the SCD group demonstrated higher preoperative (9.1 vs. 3.8; P < .001) and postoperative VAS scores (3.8 vs. 1.3; P < .001). Other clinical outcomes demonstrated no significant differences. There were 5 (29%) complications, 2 (11.8%) episodes of sickle cell crisis, and 3 (18%) reoperations in the SCD cohort. When compared to the matched cohort, there were no statistical differences with respect to complications (29% vs. 12%; P = .140) or reoperations (18% vs. 12%; P = .387). CONCLUSIONS: SA is an effective treatment modality for glenohumeral AVN in patients with SCD. Patients may expect improvements in pain, function, motion, and strength. However, final postoperative pain relief may be less than those treated with SA without SCD. Unique perioperative management is necessary to mitigate the risk of postoperative sickle cell crises.

Considerations in the Sickle Cell Patient Undergoing Hip Reconstructive Surgery.

Sickle cell disease (SCD) is a hemoglobinopathy that commonly has musculoskeletal effects including osteonecrosis of major joints (most often the hip) and medullary infarcts with resultant pain, functional limitations, and decreased quality of life. Patients with SCD may require surgical intervention, including total hip arthroplasty, frequently at a young age. The underlying pathologic process of SCD creates unique medical and surgical challenges that place these patients at increased risk of complications. This necessitates a multidisciplinary approach for providing surgical care to patients with SCD.

Simple open-heart surgery protocol for sickle-cell disease patients: a retrospective cohort study comparing patients undergoing mitral valve surgery.

OBJECTIVES: Sickle-cell disease (SCD) patients are considered to be at high risk from open-heart surgery. This study assessed the role of a simple sickling-prevention protocol. METHODS: Perioperative non-specific and SCD-specific morbidity and 30-day mortality are investigated in a retrospective cohort study on patients undergoing isolated mitral valve surgery. Patients with and without SCD were compared. In the SCD cohort, a bundle of interventions was applied to limit the risk of sickling: 'on-demand' transfusions to keep haemoglobin levels of around 7-8 g/dl, cardiopulmonary bypass (CPB) with higher blood flow and perfusion temperature, close monitoring of acid-base balance and oxygenation. RESULTS: Twenty patients with and 40 patients without SCD were included. At baseline, only preoperative haemoglobin levels differed between cohorts (8.1 vs 11.8 g/dl, P < 0.001). Solely SCD patients received preoperative transfusions (45.0%). Intraoperative transfusions were significantly larger in SCD patients during CPB (priming: 300 vs 200 ml; entire length: 600 vs 300 ml and 20 vs 10 ml/kg). SCD patients had higher perfusion temperatures during CPB (34.7 vs 33.0°C, P = 0.01) with consequently higher pharyngeal temperature, both during cooling (34.1 vs 32.3°C, P = 0.02) and rewarming (36.5 vs 36.2°C, P = 0.02). No mortality occurred, and non-SCD-specific complications were comparable between groups, but one SCD patient suffered from perioperative cerebrovascular accident with seizures, and another had evident haemolysis. CONCLUSIONS: SCD patients may undergo open-heart surgery for mitral valve procedures with an acceptable risk profile. Simple but thoughtful perioperative management, embracing 'on-demand' transfusions and less-aggressive CPB cooling is feasible and probably efficacious.

Liver Transplantation After Hematopoietic Stem Cell Transplant for the Treatment of Sickle Cell Disease: A Case Report.

Sickle cell anemia is the most common of the hemoglobinopathies, in which the abnormal hemoglobin formed in deoxygenation states undergoes a polymerization process with consequent erythrocyte deformation and vaso-occlusive events. The need for multiple blood transfusions, prolonged ineffective erythropoiesis, hemolysis, and increased iron absorption can cause iron overload in the liver, leading to liver fibrosis. Hematopoietic stem cell transplantation (HSCT) is currently the only treatment with a curative potential for this disease and can establish normal complete or partial donor-derived erythropoiesis and stabilize or restore function in affected organs, preventing further deterioration of function. However, it does not reverse preexisting liver fibrosis and siderosis. One of the possible complications of patients who undergo HSCT is chronic liver disease, which has a multifactorial cause, with iron overload being an important factor. In the long term, the prevalence of chronic liver disease in HSCT patients, including cirrhosis and its complications, can be significant. Solid organ transplantation after allogeneic hematopoietic cell transplantation for end-organ failure remains a very rare event. It may offer a valuable treatment strategy in selected recipients, although it is associated with significant morbidity and mortality. We report the case of a patient with sickle cell anemia who underwent HSCT and developed severe liver dysfunction requiring liver transplantation 13 years after the procedure. We found no previous report in the literature of orthotopic liver transplant after HCT for the treatment of sickle cell disease.

Guideline on the peri-operative management of patients with sickle cell disease: Guideline from the Association of Anaesthetists.

Sickle cell disease is a multisystem disease characterised by chronic haemolytic anaemia, painful vaso-occlusive crises and acute and chronic end-organ damage. It is one of the most common serious inherited single gene conditions worldwide and has a major impact on the health of affected individuals. Peri-operative complications are higher in patients with sickle cell disease compared with the general population and may be sickle or non-sickle-related. Complications may be reduced by meticulous peri-operative care and transfusion, but unnecessary transfusion should be avoided, particularly to reduce the risk of allo-immunisation. Planned surgery and anaesthesia for patients with sickle cell disease should ideally be undertaken in centres with experience in caring for these patients. In an emergency, advice should be sought from specialists with experience in sickle cell disease through the haemoglobinopathy network arrangements. Emerging data suggest that patients with sickle cell disease are at increased risk of COVID-19 infection but may have a relatively mild clinical course. Outcomes are determined by pre-existing comorbidities, as for the general population.

[Allogeneic hematopoietic stem cell transplant in children and adults with sickle cell disease: Indications and modalities]. / Allogreffe de cellules souches hématopoïétiques dans la drépanocytose de l'enfant et de l'adulte : indications et modalités.

Sickle cell disease is associated with severe complications and early mortality in adults. In children, hematopoietic stem cell transplant from HLA-identical sibling can stop the progression of the disease and leads to more than 95% long-term free survival without sickle cell disease. The aim of this workshop was to define indications and modalities of allogeneic hematopoietic stem cell transplant in children and adults with sickle cell disease. Patient and sibling HLA typing should be proposed, early in the course of the disease, when intensification therapies are required. Indications of transplant from HLA-identical sibling in children and adults are, cerebral vasculopathy, occurrence of vaso-occlusive events despite hydroxycarbamide, renal and hepatic diseases related to SCD, chronic anemia<7g/dL despite hydroxycarbamide, need to maintain transfusion programs longer than six months, and major transfusion difficulties related to red blood cell alloimmunization. In children with an HLA-identical sibling donor, we recommend a myeloablative conditioning regimen associating high dose busulfan, cyclophosphamide and ATG, considering the excellent results of this approach In patients over 15 years of age, we recommend the NIH approach consisting of a reduced intensity conditioning regimen by alemtuzumab, and 3Gy total body irradiation, followed by peripheral hematopoietic stem cells and post-transplant immunosuppression by sirolimus In the absence of HLA-identical sibling donor, there is no definitive data for preferring transplant from unrelated versus haplo-identical donors but we recommend to evaluate these approaches in prospective trials.

Preoperative blood transfusions for sickle cell disease.

BACKGROUND: Sickle cell disease (SCD) is one of the commonest severe monogenic disorders in the world, due to the inheritance of two abnormal haemoglobin (beta globin) genes. SCD can cause severe pain, significant end-organ damage, pulmonary complications, and premature death. Surgical interventions are more common in people with SCD, and occur at much younger ages than in the general population. Blood transfusions are frequently used prior to surgery and several regimens are used but there is no consensus over the best method or the necessity of transfusion in specific surgical cases. This is an update of a Cochrane Review. OBJECTIVES: To determine whether there is evidence that preoperative blood transfusion in people with SCD undergoing elective or emergency surgery reduces mortality and perioperative or sickle cell-related serious adverse events. To compare the effectiveness of different transfusion regimens (aggressive or conservative) if preoperative transfusions are indicated in people with SCD. SEARCH METHODS: We searched for relevant trials in the Cochrane Library, MEDLINE (from 1946), Embase (from 1974), the Transfusion Evidence Library (from 1980), and ongoing trial databases; all searches current to 28 January 2020 We searched the Cochrane Cystic Fibrosis and Genetic Disorders Group Trials Register: 19 September 2019. SELECTION CRITERIA: All randomised controlled trials and quasi-randomised controlled trials comparing preoperative blood transfusion regimens to different regimens or no transfusion in people with SCD undergoing elective or emergency surgery. There was no restriction by outcomes examined, language or publication status. DATA COLLECTION AND ANALYSIS: Two authors independently assessed trial eligibility and the risk of bias and extracted data. MAIN RESULTS: Three trials with 990 participants were eligible for inclusion in the review. There were no ongoing trials identified. These trials were conducted between 1988 and 2011. The majority of people included had haemoglobin (Hb) SS SCD. The majority of surgical procedures were considered low or intermediate risk for developing sickle cell-related complications. Aggressive versus simple red blood cell transfusions One trial (551 participants) compared an aggressive transfusion regimen (decreasing sickle haemoglobin to less than 30%) to a simple transfusion regimen (increasing haemoglobin to 100 g/L). This trial re-randomised participants and therefore quantitative analysis was only possible on two subsets of data: participants undergoing cholecystectomy (230 participants); and participants undergoing tonsillectomy or adenoidectomy surgeries (107 participants). Data were not combined as we do not know if any participant received both surgeries. Overall, the quality of the evidence was very low across different outcomes according to GRADE methodology. This was due to the trial being at high risk of bias primarily due to lack of blinding, indirectness and the outcome estimates being imprecise. Cholecystectomy subgroup results are reported in the abstract. Results for both subgroups were similar. There was no difference in all-cause mortality between people receiving aggressive transfusions and those receiving conservative transfusions. No deaths occurred in either subgroup. There were no differences between the aggressive transfusion group and conservative transfusion group in the number of people developing: • an acute chest syndrome, risk ratio (RR) 0.84 (95% confidence interval (CI) 0.38 to 1.84) (one trial, 230 participants, very low-quality evidence); • vaso-occlusive crisis, risk ratio 0.30 (95% CI 0.09 to 1.04) (one trial, 230 participants, very low quality evidence); • serious infection, risk ratio 1.75 (95% CI 0.59 to 5.18) (one trial, 230 participants, very low-quality evidence); • any perioperative complications, RR 0.75 (95% CI 0.36 to 1.55) (one trial, 230 participants, very low-quality evidence); • a transfusion-related complication, RR 1.85 (95% CI 0.89 to 3.88) (one trial, 230 participants, very low-quality evidence). Preoperative transfusion versus no preoperative transfusion Two trials (434 participants) compared a preoperative transfusion plus standard care to a group receiving standard care. Overall, the quality of the evidence was low to very low across different outcomes according to GRADE methodology. This was due to the trials being at high risk of bias due to lack of blinding, and outcome estimates being imprecise. One trial was stopped early because more people in the no transfusion arm developed an acute chest syndrome. There was no difference in all-cause mortality between people receiving preoperative transfusions and those receiving no preoperative transfusions (two trials, 434 participants, no deaths occurred). There was significant heterogeneity between the two trials in the number of people developing an acute chest syndrome, a meta-analysis was therefore not performed. One trial showed a reduced number of people developing acute chest syndrome between people receiving preoperative transfusions and those receiving no preoperative transfusions, risk ratio 0.11 (95% confidence interval 0.01 to 0.80) (65 participants), whereas the other trial did not, RR 4.81 (95% CI 0.23 to 99.61) (369 participants). There were no differences between the preoperative transfusion groups and the groups without preoperative transfusion in the number of people developing: • a vaso-occlusive crisis, Peto odds ratio (OR) 1.91 (95% confidence interval 0.61 to 6.04) (two trials, 434 participants, very low-quality evidence). • a serious infection, Peto OR 1.29 (95% CI 0.29 to 5.71) (two trials, 434 participants, very low-quality evidence); • any perioperative complications, RR 0.24 (95% CI 0.03 to 2.05) (one trial, 65 participants, low-quality evidence). There was an increase in the number of people developing circulatory overload in those receiving preoperative transfusions compared to those not receiving preoperative transfusions in one of the two trials, and no events were seen in the other trial (no meta-analysis performed). AUTHORS' CONCLUSIONS: There is insufficient evidence from randomised trials to determine whether conservative preoperative blood transfusion is as effective as aggressive preoperative blood transfusion in preventing sickle-related or surgery-related complications in people with HbSS disease. There is very low quality evidence that preoperative blood transfusion may prevent development of acute chest syndrome. Due to lack of evidence this review cannot comment on management for people with HbSC or HbSß+ disease or for those with high baseline haemoglobin concentrations.

Hematopoietic stem cell transplantation for people with sickle cell disease.

BACKGROUND: Sickle cell disease is a genetic disorder involving a defect in the red blood cells due to its sickled hemoglobin. The main therapeutic interventions include preventive and supportive measures. Hematopoietic stem cell transplantations are carried out with the aim of replacing the defective cells and their progenitors (hematopoietic (i.e. blood forming) stem cells) in order to correct the disorder. This is an update of a previously published review. OBJECTIVES: To determine whether stem cell transplantation can improve survival and prevent symptoms and complications associated with sickle cell disease. To examine the risks of stem cell transplantation against the potential long-term gain for people with sickle cell disease. SEARCH METHODS: We searched the Cochrane Cystic Fibrosis and Genetic Disorders Group Group's Haemoglobinopathies Trials Register complied from electronic searches of the Cochrane Central Register of Controlled Trials (CENTRAL) (updated each new issue of the Cochrane Library) and quarterly searches of MEDLINE. We also searched trial registries for ongoing trials up to April 2020. Date of the most recent search of the Group's Haemoglobinopathies Trials Register: 09 December 2019. SELECTION CRITERIA: Randomized controlled and quasi-randomized trials that compared any method of stem cell transplantation with either each other or with any of the preventive or supportive interventions (e.g. periodic blood transfusion, use of hydroxyurea, antibiotics, pain relievers, supplemental oxygen) in people with sickle cell disease irrespective of the type of sickle cell disease, gender and setting. DATA COLLECTION AND ANALYSIS: No trials were eligible for inclusion in the review. MAIN RESULTS: We identified 12 potentially-eligible trials by the searches; we excluded 11 of these and the remaining trial is an ongoing trial that may be eligible for inclusion in a future version of the review. AUTHORS' CONCLUSIONS: Reports on the use of hematopoietic stem cell transplantation improving survival and preventing symptoms and complications associated with sickle cell disease are currently limited to observational and other less robust studies. We did not find any eligible randomized controlled trials assessing the benefit or risk of hematopoietic stem cell transplantations. However, there is an ongoing quasi-randomized trial comparing hematopoietic stem cell transplantation with standard care, Thus, this systematic review identifies the need for a multicentre randomized controlled trial assessing the benefits and possible risks of hematopoietic stem cell transplantations comparing sickle status and severity of disease in people with sickle cell disease.

Assessment of Patient and Caregiver Attitudes and Approaches to Decision-Making Regarding Bone Marrow Transplant for Sickle Cell Disease: A Qualitative Study.

Importance: Bone marrow transplant (BMT) is a potentially curative treatment for sickle cell disease (SCD). Patient and caregiver attitudes toward BMT for SCD and the willingness to accept risks of BMT vary, but these attitudes are not well understood. Objective: To understand patient and caregiver perceptions of and attitudes toward BMT for SCD and decision-making about BMT. Design, Setting, and Participants: Qualitative study of interview transcripts from a convenience sample. Transcripts were from adults with SCD and caregivers of patients with SCD recruited from national and regional SCD conferences, symposia, and sickle cell clinics in 2 cities. Interview transcripts were used from the needs assessment phase to develop a patient-decision aid in 2013 to 2014 (group 1) and from the baseline point in 2015 to 2016 (group 2) of the parent trial, a randomized clinical trial of adults and caregivers of patients with SCD to evaluate the effectiveness of a patient decision aid. Main Outcomes and Measures: Participant perspectives on decision-making regarding BMT for SCD. Results: Fifty-seven transcripts from adults with SCD and 50 transcripts from caregivers of patients with SCD were included. Median (interquartile range [IQR]) age of adults with SCD was 34 (21-50) years in group 1 and 30 (23-38) years in group 2. The median (IQR) age of caregivers was 42.5 (31-52) years in group 1 and 41 (35-46.5) years in group 2. Most transcripts from adults with SCD (75.0% in group 1 and 72.4% in group 2) and caregivers of patients with SCD (76.7% in group 1 and 85.0% in group 2) were from female participants. Bone marrow transplant was perceived as a treatment option associated with serious risks. Reported attitudes toward BMT occurred on a continuum ranging from unfavorable to favorable. Participants reported serious decisional dilemma regarding BMT for SCD. Most participants expressed interest in learning about BMT or curative treatments. Conclusions and Relevance: This qualitative study found a continuum in attitudes toward BMT for SCD and highlights the complexity of decision-making in BMT for SCD. Patients and families with SCD expressed an interest in learning about BMT. Future prospective studies of patient decision-making regarding BMT, especially in the context of emerging curative and novel disease-modifying therapies for SCD, are warranted.

Total Hip Arthroplasty in Patients With Sickle Cell Disease: A Comprehensive Systematic Review.

BACKGROUND: Total hip arthroplasty (THA) is a common treatment for end-stage osteonecrosis of the hip in patients with sickle cell disease (SCD). This patient population presents unique challenges in the perioperative period. This systematic review aims to investigate the existing literature on the outcomes, complications, and survivorship of primary THA in SCD patients. METHODS: A systematic search using Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines was performed of PubMed, MEDLINE, EMBASE, and Cochrane databases for clinical studies on THA in SCD patients. Studies on primary THA in SCD patients with a mean follow-up greater than 90 days were included. RESULTS: Sixteen studies containing 5193 SCD patients met criteria for inclusion. The Coleman Quality of Evidence score ranged from poor to moderate. SCD patients had a significant increase in Harris Hip Scores and Merle d'Aubigne Scores after undergoing THA. Compared to non-SCD patients, SCD patients had increased hospital length-of-stay, 30-day and 90-day readmission rates, and rates of medical complications, including pain crises, acute chest syndrome, cardiac complications, sepsis, and mortality. SCD patients also had increased rates of surgical complications, including wound complications, infection, periprosthetic fracture, and aseptic loosening. Overall, THA revision rates were higher in SCD patients relative to those with primary osteoarthritis. CONCLUSION: THA remains an effective treatment modality for osteonecrosis of the hip in SCD patients. However, these patients are at increased risk of medical and surgical complications. Surgeons should be aware of the unique challenges in this patient population when counseling and managing these patients in the perioperative period.

Outcomes of a preoperative risk-based transfusion assignment protocol in sickle cell disease patients: a single-center retrospective study from Saudi Arabia.

Many patients with sickle cell disease (SCD) need surgical management during their lifetime. The best approach for preoperative transfusion in SCD is still to be determined. In this single-center retrospective study, we included HBSS/HBS-Beta0-thalassemia patients younger than 16 years of age who underwent surgery between January 2008 and July 2019. Preoperative transfusion assignment (PTA) was based on SCD severity and surgical risk. Patients were assigned to no transfusion, simple transfusion, or exchange transfusion. A total of 284 patients were identified and 66 (23%) underwent 78 procedures. Mean age at the time of procedure was 8 (5-11) years, mean baseline hemoglobin was 8.5 (7.8-9.3) g/dl, and mean hemoglobin F was 18.4 ± 8.2%. SCD severity was low-risk in 57 (73%) and high-risk in 21 (27%) patients. Surgical risk was low-risk in 20 (25.6%) and medium-risk in 58 (74.4%) procedures. PTA was no transfusion in 17 (22%), simple transfusion in 40 (51%), and exchange transfusion in 21 (27%) procedures. Postoperative complications occurred in five (6.4%) of procedures only in the simple transfusion group (three acute chest syndrome, one hemolytic anemia, one pain crisis) undergoing medium-risk surgery. Preoperative risk-based transfusion assignment is feasible. Despite a high baseline hemoglobin level in the no transfusion group, none of the patients developed postoperative complications. It is possible that the high baseline hemoglobin F phenotype was protective and indicates the need to study the risk/benefit of interventions used in this phenotype.