RESUMO
Isothermal nucleic acid amplification tests have the potential to improve disease diagnosis at the point of care, but it remains challenging to develop multiplexed tests that can detect ≥3 targets or to detect point mutations that may cause disease. These capabilities are critical to enabling informed clinical decision-making for many applications, such as sickle cell disease (SCD). To address this, we describe the development of a multiplexed allele-specific recombinase polymerase amplification (RPA) assay with lateral flow readout. We first characterize the specificity of RPA using primer design strategies employed in PCR to achieve point mutation detection, and demonstrate the utility of these strategies in achieving selective isothermal amplification and detection of genomic DNA encoding for the healthy ßA globin allele, or genomic DNA containing point mutations encoding for pathologic ßS and ßC globin alleles, which are responsible for most sickle cell disorders. We then optimize reaction conditions to achieve multiplexed amplification and identification of the three alleles in a single reaction. Finally, we perform a small pilot study with 20 extracted genomic DNA samples from SCD patients and healthy volunteers - of the 13 samples with valid results, the assay demonstrated 100% sensitivity and 100% specificity for detecting pathologic alleles, and an overall accuracy of 92.3% for genotype prediction. This multiplexed assay is rapid, minimally instrumented, and when combined with point-of-care sample preparation, could enable DNA-based diagnosis of SCD in low-resource settings. The strategies reported here could be applied to other challenges, such as detection of mutations that confer drug resistance.
Assuntos
Alelos , Anemia Falciforme , Técnicas de Amplificação de Ácido Nucleico , Recombinases , Anemia Falciforme/genética , Anemia Falciforme/diagnóstico , Humanos , Recombinases/metabolismo , Globinas beta/genética , Mutação PuntualRESUMO
Sickle cell disease (SCD) and systemic lupus erythematosus (SLE) are two uncommon disorders each characterized by multisystemic manifestations. Individuals with SCD exhibit abnormalities in the complement pathway, which may predispose patients to develop autoimmune disorders such as SLE. As many manifestations of SLE mimic those of SCD, diagnosis and therapeutic management of SLE in a patient with known SCD may be delayed. In this study, we describe our institutional experience of diagnosing and managing concomitant SCD and SLE. We offer insights into the complex interplay between these conditions to enhance early recognition and effective management of concurrent SCD and SLE.
Assuntos
Anemia Falciforme , Lúpus Eritematoso Sistêmico , Adolescente , Feminino , Humanos , Adulto Jovem , Anemia Falciforme/complicações , Lúpus Eritematoso Sistêmico/complicaçõesRESUMO
A 4-month-old full-term female presented with growth faltering associated with progressive feeding difficulty, rash, abdominal distension, and developmental delays. She was found to have disconjugate gaze, abnormal visual tracking, mixed tone, bruising, and splenomegaly on examination. Initial workup was notable for thrombocytopenia and positive cytomegalovirus (CMV) immunoglobulin G and immunoglobulin M antibodies. She initially presented to the infectious diseases CMV clinic, where she was noted to have severe malnutrition, prompting referral to the emergency department for hospital admission to optimize nutrition with nasogastric tube feeding and facilitate additional evaluation. An active CMV infection with viruria and viremia was confirmed, but elements of her presentation and workup including brain magnetic resonance imaging were not consistent with isolated CMV infection. To avoid premature diagnostic closure, a multidisciplinary workup was initiated and ultimately established her diagnosis.
Assuntos
Esplenomegalia , Trombocitopenia , Humanos , Feminino , Lactente , Esplenomegalia/etiologia , Esplenomegalia/diagnóstico por imagem , Trombocitopenia/diagnóstico , Infecções por Citomegalovirus/complicações , Infecções por Citomegalovirus/diagnóstico , Insuficiência de Crescimento/etiologia , Diagnóstico DiferencialRESUMO
Early mortality in sickle cell disease (SCD) is attributed to increased infections due to loss of splenic function. Marginal zone B cells are important for initial opsonization of pathogens and can be absent in spleen histopathology in SCD. The frequency of unswitched memory B cells (UMBC), the circulating correlate of marginal zone B cells, reflects the immunologic function of the spleen. We hypothesized that asplenia in SCD is associated with alterations in the peripheral blood lymphocyte population and explored whether UMBC deficiency was associated with a clinical phenotype. We analyzed B cell subsets and clinical history for 238 children with SCD and 63 controls. The median proportion of UMBCs was lower in children with SCD compared with controls (4.7% vs. 6.6%, p < .001). Naïve B cells were higher in SCD compared with controls (80.6 vs. 76.3%, respectively, p = .02). UMBC frequency declined by 3.4% per year increase in age in SCD (95% CI: 2%, 4.7%, p < .001), but not in controls. A majority of children in all cohorts had an IgM concentration in the normal range for age and there were no differences between groups (p = .13). Subjects developed titers adequate for long-term protection to fewer serotypes in the polysaccharide vaccine than controls (14.7 vs. 19.4, p < .001). In this cohort, bacteremia was rare and specific clinical complications were not associated with UMBC proportion. In summary, UMBC deficiency occurs in SCD and is associated with age. Future studies should investigate B cell subsets prospectively and identify the mechanism of B cell loss in the spleen.
Assuntos
Anemia Falciforme , Células B de Memória , Vacinas Pneumocócicas , Humanos , Anemia Falciforme/imunologia , Anemia Falciforme/complicações , Vacinas Pneumocócicas/imunologia , Vacinas Pneumocócicas/uso terapêutico , Criança , Masculino , Feminino , Pré-Escolar , Células B de Memória/imunologia , Adolescente , Subpopulações de Linfócitos B/imunologia , Infecções Pneumocócicas/imunologia , Infecções Pneumocócicas/prevenção & controle , Baço/imunologia , Baço/patologia , Imunoglobulina M/sangueRESUMO
Crizanlizumab, a monoclonal antibody against P-selectin, has been shown to reduce vaso-occlusive crises (VOCs) compared to placebo in patients ≥ 16 years with sickle cell disease (SCD). However, there have been rare reports of patients experiencing severe pain and subsequent complications within 24 hours of crizanlizumab infusions. These events are defined as infusion-related reactions (IRRs). Informed by current literature and clinical experience, a group of content experts developed clinical guidelines for the management of IRRs in patients with SCD. We used the RAND/University of California, Los Angeles (UCLA) modified Delphi panel method, a valid, reproducible technique for achieving consensus. We present our recommendations for managing IRRs, which depend on patient characteristics including: prior history of IRRs to other monoclonal antibodies or medications, changes to crizanlizumab infusion rate and patient monitoring, pain severity relative to patient's typical SCD crises, and severe allergic symptoms. These recommendations outline how to evaluate and manage IRRs in patients receiving crizanlizumab. Future research should validate this guidance using clinical data and identify patients at risk for these IRRs.
Assuntos
Anemia Falciforme , Anticorpos Monoclonais Humanizados , Técnica Delphi , Humanos , Anticorpos Monoclonais Humanizados/uso terapêutico , Anticorpos Monoclonais Humanizados/efeitos adversos , Anemia Falciforme/tratamento farmacológico , Infusões Intravenosas , ConsensoRESUMO
CONTEXT: Pain attributable to sickle cell disease (SCD) is often unpredictable, recurrent, and requires complex treatments. Subanesthetic ketamine infusion has been studied in other diseases and disorders, but there is still limited data on its efficacy in pain management for SCD. OBJECTIVES: The primary objective is to determine if subanesthetic ketamine infusion reduces pain scores and opioid requirements in hospitalized pediatric patients with SCD. RESULTS: Forty-six admissions among 22 patients between February 2018 and December 2019 were analyzed. We observed decrease in pain scores within 24 hours of ketamine initiation in 34 of 46 admissions (mean pain score per patient before ketamine initiation: 2.2-9.7, mean pain score per patient after ketamine initiation: 0-9.7; P < .05). We observed a decrease in pain scores in the remaining 12 admissions after greater than 24 hours of ketamine initiation. Opioid usage declined after ketamine infusion, with a difference of means in oral morphine equivalents before and after ketamine of 122.8 mg/day. The side effects observed with ketamine infusion included hallucinations in 11 (23.9%) admissions. Only four (8.7%) admissions required cessation of the infusion due to side effects. The readmission rate at two weeks and four weeks after first ketamine infusion was the same (12.5%) at both time points. For all patients in the cohort, the introduction of ketamine into pain regimens did not reduce the number of admissions in the year following ketamine initiation relative to the year prior. CONCLUSION: In pediatric patients with SCD, subanesthetic ketamine was safe as a continuous infusion and effectively reduced both pain scores and opioid requirements.
Assuntos
Anemia Falciforme , Ketamina , Humanos , Criança , Ketamina/uso terapêutico , Ketamina/efeitos adversos , Analgésicos Opioides/uso terapêutico , Dor/tratamento farmacológico , Dor/etiologia , Morfina , Anemia Falciforme/complicações , Anemia Falciforme/tratamento farmacológico , AnalgésicosRESUMO
OBJECTIVE: Voxelotor, a FDA-approved drug for the treatment of patients with sickle cell disease (SCD), inhibits hemoglobin S (HbS) polymerization and increases total hemoglobin via hemolysis reduction. This drug has shown unique patterns in hemoglobin fractionation, affecting its interpretation. We aimed to evaluate whether these voxelotor-induced changes can be linked to improvement of hemolysis markers in pediatric patients on voxelotor. METHODS: A total of 15 patients (age 12 to 20 years; 40% females) on voxelotor were evaluated to compare changes in the hemoglobin fractionation by capillary electrophoresis, total hemoglobin, reticulocyte percentage (retic%), lactate dehydrogenase (LDH), and bilirubin measurements before and after the recorded date of voxelotor prescription. RESULTS: Hemoglobin fractionation showed changes in the profile of 60% (9/15) of the patients studied. Out of the 9 patients for which voxelotor showed changes in the hemoglobin fractionation, 44% (4/9) had an increase of >1 g/dL in their total hemoglobin after voxelotor treatment was started. Assessment of other hemolysis markers available showed decreased LDH (4/4), retic % (6/8), and bilirubin (3/4). CONCLUSIONS: Unique pattern of hemoglobin fractionation analysis following therapy with voxelotor has potential as a tool for the assessment of response and/or compliance to voxelotor for the treatment of SCD.
Assuntos
Anemia Falciforme , Hemoglobinopatias , Feminino , Humanos , Criança , Adolescente , Adulto Jovem , Adulto , Masculino , Hemólise , Hemoglobinopatias/tratamento farmacológico , Anemia Falciforme/tratamento farmacológico , BilirrubinaRESUMO
Because of the unique biology of sickle cell disease (SCD) as well as the societal disadvantages and racial inequities suffered by these patients, individuals with SCD have not benefited from the same remarkable advances in care and therapeutics as those with other hematologic disorders. Life expectancy of individuals with SCD is shortened by â¼20 years even with optimal clinical care, and infant mortality continues to be a major concern in low-income countries. As hematologists, we must do more. The American Society of Hematology (ASH) and the ASH Research Collaborative have instituted a multipronged initiative to improve the lives of individuals living with this disease. Here, we describe 2 components of this ASH initiative, the Consortium on Newborn Screening in Africa (CONSA) to improve the early diagnosis of infants in low-resource countries and the SCD Clinical Trial Network to accelerate the development of more effective therapeutics and care for those with this disorder. The combination of SCD-focused initiatives, ASH Research Collaborative, CONSA, and Sickle Cell Clinical Trials Network has enormous potential to dramatically alter the course of SCD worldwide. We believe that the timing is ripe to embark on these critical and worthwhile initiatives and improve the lives of individuals with this disease.
Assuntos
Anemia Falciforme , Doenças Hematológicas , Lactente , Recém-Nascido , Humanos , Anemia Falciforme/terapia , Anemia Falciforme/tratamento farmacológico , Expectativa de Vida , Assistência ao Paciente , Triagem NeonatalRESUMO
Data on outcomes and interventions for children with sickle cell disease (SCD) admitted to a pediatric intensive care units (PICU) are unknown. We provide the first comprehensive multi-center report on PICU interventions associated with death, the need for invasive respiratory support or stroke among critically ill children with SCD. We collected retrospective multi-center cohort data from January 1, 2012 to December 31, 2019 utilizing the Virtual Pediatric Systems, LLC database. We identified 3388 unique children with SCD, accounting for a total of 5264 PICU admissions from 138 PICUs. The overall mortality rate for the PICU admissions cohort was 1.8% (95/5264 PICU admissions, 95/3388 [2.8%] of all unique patients), the rate of needing of needing Invasive Respiratory Support (IRS, a composite category of exposure) was 21.3% (872/4093 PICU admissions with complete data) and the overall rate of stroke (ischemic or hemorrhagic) was 12.5% (657/5264 PICU admissions). In multivariable analysis adjusting for admission age category, sex, race/ethnicity, PRISM-3 score at admission, exposure to IRS, quartile of unit volume of patients with SCD, and patient origin, admitted children who needed invasive respiratory support (IRS) had higher adjusted odds ratios for mortality (adjusted odds ratio [aOR], 19.72; 95% confidence interval [CI] 8.98-43.29; p < 0.001), although admitted children > 2 years old had decreased aOR for needing IRS (aOR 0.25-0.62; 95% CI 0.16-0.94; p < 0.001-0.025). By contrast, admitted children > 2 years old had a strikingly increased aOR for stroke (aOR 7.57-16.32; 95% CI 2.25-52.15; p < 0.001). These groups may represent PICU-specific subsets of patients with SCD who are at higher risk for more serious illness and should deserve early consideration for referral to a pediatric institution providing comprehensive care for patients with SCD.
Assuntos
Anemia Falciforme , Acidente Vascular Cerebral , Humanos , Criança , Estados Unidos/epidemiologia , Lactente , Pré-Escolar , Estudos Retrospectivos , Mortalidade Hospitalar , Unidades de Terapia Intensiva Pediátrica , Anemia Falciforme/epidemiologia , Anemia Falciforme/terapiaRESUMO
High-income nations have established that early diagnosis and preventive treatment reduces early deaths in sickle cell disease (SCD). However, in low-/middle-income countries where SCD is common, attrition from clinical care is common. Reasons for poor retention in care are multi-factorial and poorly understood. The objective of this study was to identify factors that influence caregiver decision-making around chronic health care needs of a child with SCD. We conducted an exploratory sequential mixed methods study of caregivers of children diagnosed with SCD during a newborn screening program in Liberia. Caregivers completed questionnaires and semi-structured interviews designed to identify drivers of health decision-making. Interviews were digitally recorded, transcribed, coded, and analyzed using semi-structured thematic analysis to identify themes. Data integration occurred by using quantitative results to expand and clarify the qualitative themes. Twenty-six caregivers participated in the study. The mean age of the child at the interview was 43.7 months. Five themes influencing health decisions were identified: grief, the importance of support networks, stigma, perceived benefits, and the burden of chronic disease. The five themes crossed multiple domains of a socioecological model and identified complex interactions between family, community, social and cultural norms, and organizational structures. This study highlights the importance of community awareness of SCD and appropriate health communication by healthcare workers. Healthcare decision-making is multifactorial and complex. These results provide a framework for improving retention in care. In a low-resource country such as Liberia, much can be done by leveraging existing resources and cultural practices.
RESUMO
Sickle cell disease (SCD) is a common condition within sub-Saharan Africa and associated with high under-5 mortality (U5M). The American Society of Hematology instituted the Consortium on Newborn Screening in Africa (CONSA) for SCD, a 7-country network of sites to implement standardized newborn hemoglobinopathy screening and early intervention for children with SCD in sub-Saharan Africa. CONSA's overall hypothesis is that early infant SCD screening and entry into standardized, continuous care will reduce U5M compared with historical estimates in the region. Primary trial objectives are to determine the population-based birth incidence of SCD and effectiveness of early standardized care for preventing early mortality consortium-wide at each country's site(s). Secondary objectives are to establish universal screening and early interventions for SCD within clinical networks of CONSA partners and assess trial implementation. Outcomes will be evaluated from data collected using a shared patient registry. Standardized trial procedures will be implemented among designated birth populations in 7 African countries whose programs met eligibility criteria. Treatment protocol includes administering antibacterial and antimalarial prophylaxis and standard childhood vaccinations against infections commonly affecting children with SCD. Infants with a positive screen and confirmation of SCD within the catchment areas defined by each consortium partner will be enrolled in the clinical intervention protocol and followed regularly until age of 5 years. Effectiveness of these early interventions, along with culturally appropriate family education and counseling, will be evaluated by comparing U5M in the enrolled cohort to estimated preprogram data. Here, we describe the methodology planned for this trial.
Assuntos
Anemia Falciforme , Triagem Neonatal , Lactente , Criança , Recém-Nascido , Humanos , Pré-Escolar , Triagem Neonatal/métodos , Anemia Falciforme/diagnóstico , Anemia Falciforme/epidemiologia , Anemia Falciforme/complicações , África Subsaariana/epidemiologia , IncidênciaRESUMO
PURPOSE OF REVIEW: Low-income and middle-income countries (LMICs), primarily in sub-Saharan Africa (SSA), predominantly experience the burden of sickle cell disease (SCD). High frequency of acute and chronic complications leads to increased utilization of healthcare, which burdens fragile health systems. Mortality for children with limited healthcare access remains alarmingly high. Cellular based therapies such as allogeneic hematopoietic stem cell transplant (HSCT) are increasingly used in resource-rich settings as curative therapy for SCD. Broad access to curative therapies for SCD in SSA would dramatically alter the global impact of the disease. RECENT FINDINGS: Currently, application of cellular based therapies in LMICs is limited by cost, personnel, and availability of HSCT-specific technologies and supportive care. Despite the challenges, HSCT for SCD is moving forward in LMICs. Highly anticipated gene modification therapies have recently proven well tolerated and feasible in clinical trials in resource-rich countries, but access remains extremely limited. SUMMARY: Translation of curative cellular based therapies for SCD should be prioritized to LMICs where the disease burden and cost of noncurative treatments is high, and long-term quality of life is poor. Focus on thoughtful modifications of current and future therapies to meet the need in LMICs, especially in SSA, will be especially impactful.
Assuntos
Anemia Falciforme , Doença Enxerto-Hospedeiro , Transplante de Células-Tronco Hematopoéticas , África Subsaariana/epidemiologia , Anemia Falciforme/complicações , Anemia Falciforme/terapia , Criança , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Humanos , Qualidade de VidaRESUMO
Pain management is challenging for patients with sickle cell disease (SCD) who present in vaso-occlusive crisis (VOC). Opioid therapy is highly effective, nevertheless undesirable side effects can hinder their effectiveness. Regional anesthesia with deposition of perineural anesthetic offers nociceptive blockade, local vasodilatation, and reduces the inflammatory response. Among pediatric patients, continuous peripheral nerve block (CPNB) for perioperative adjunctive analgesia is safe. Herein, we describe the trajectory of a cohort of pediatric SCD patients with opioid-refractory upper-extremity VOC following placement of CPNBs for analgesia; highlighting reduced opioid consumption, improved pain scores, and decreased length of hospitalization.
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Anemia Falciforme , Anestesia por Condução , Analgésicos Opioides/uso terapêutico , Anemia Falciforme/complicações , Anemia Falciforme/terapia , Criança , Humanos , Dor/tratamento farmacológico , Dor/etiologiaRESUMO
OBJECTIVES: Given the fundamental role of newborn bloodspot screening (NBS) to enable prompt diagnosis and optimal clinical management of individuals with sickle cell disease (SCD), we sought to systematically assess enablers and barriers to implementation of NBS programmes for SCD in Africa using established qualitative research methods. SETTING: Childbirth centres and NBS laboratories from six countries in East, West and Southern Africa. PARTICIPANTS: Eight programme leaders involved with establishing and operating NBS programmes for SCD in Angola, Democratic Republic of Congo, Ghana, Liberia, Nigeria and Tanzania. PRIMARY AND SECONDARY OUTCOME MEASURES: Data obtained through a structured, phased interview approach were analysed using a combination of inductive and deductive codes and used to determine primary themes related to the implementation and sustainability of SCD NBS programmes. RESULTS: Four primary themes emerged from the analysis relating to governance (eg, pragmatic considerations when deploying overcommitted clinical staff to perform NBS), technical (eg, design and execution of operational processes), cultural (eg, variability of knowledge and perceptions of community-based staff) and financial (eg, issues that can arise when external funding may effectively preclude government inputs) aspects. Key learnings included perceived factors that contribute to long-term NBS programme sustainability. CONCLUSIONS: The establishment of enduring NBS programmes is a proven approach to improving the health of populations with SCD. Organising such programmes in Africa is feasible, but initial implementation does not assure sustainability. Our analysis suggests that future programmes should prioritise government partner participation and funding from the earliest stages of programme development.
Assuntos
Anemia Falciforme , Triagem Neonatal , Anemia Falciforme/diagnóstico , Humanos , Recém-Nascido , Nigéria , Avaliação de Programas e Projetos de Saúde , Pesquisa QualitativaRESUMO
The transfusion of red blood cells (RBCs) is a crucial treatment for sickle cell disease (SCD). While often beneficial, the frequent use of transfusions is associated with numerous complications. Transfusions should be offered with specific guidelines in mind. Here we present updates to the indications for transfusion of RBCs in SCD. We review recent publications and include expert perspectives from hematology and transfusion medicine. For some clinical indications, such as ischemic stroke, the role of transfusion has been well studied and can be applied almost universally. For many other clinical scenarios, the use of transfusion therapy has less conclusive data and therefore must be tailored to individual needs. We highlight the roles of RBC transfusions in preventing or mitigating neurological disease, in reducing perioperative complications, in managing acute chest syndrome, and in optimizing pregnancy outcomes in SCD. We further highlight various transfusion techniques and when each might be considered. Potential complications of transfusion are also briefly discussed.
Assuntos
Anemia Falciforme/terapia , Transfusão de Eritrócitos/métodos , Síndrome Torácica Aguda/etiologia , Síndrome Torácica Aguda/prevenção & controle , Anemia Falciforme/complicações , Pré-Escolar , Transfusão de Eritrócitos/efeitos adversos , Feminino , Humanos , Gravidez , Complicações Hematológicas na Gravidez/terapia , Acidente Vascular Cerebral/etiologia , Acidente Vascular Cerebral/prevenção & controleRESUMO
Sickle cell disease (SCD) is a group of common, life-threatening disorders caused by a point mutation in the ß globin gene. Early diagnosis through newborn and early childhood screening, parental education, and preventive treatments are known to reduce mortality. However, the cost and complexity of conventional diagnostic methods limit the feasibility of early diagnosis for SCD in resource-limited areas worldwide. Although several point-of-care tests are commercially available, most are antibody-based tests, which cannot be used in patients who have recently received a blood transfusion. Here, we describe the development of a rapid, low-cost nucleic acid test that uses real-time fluorescence to detect the point mutation encoding hemoglobin S (HbS) in one round of isothermal recombinase polymerase amplification (RPA). When tested with a set of clinical samples from SCD patients and healthy volunteers, our assay demonstrated 100% sensitivity for both the ßA globin and ßS globin alleles and 94.7 and 97.1% specificities for the ßA globin allele and ßS globin allele, respectively (n = 91). Finally, we demonstrate proof-of-concept sample-to-answer genotyping of genomic DNA from capillary blood using an alkaline lysis procedure and direct input of diluted lysate into RPA. The workflow is performed in <30 min at a cost of <$5 USD on a commercially available benchtop fluorimeter and an open-source miniature fluorimeter. This study demonstrates the potential utility of a rapid, sample-to-answer nucleic acid test for SCD that may be implemented near the point of care and could be adapted to other disease-causing point mutations in genomic DNA.
Assuntos
Anemia Falciforme , Recombinases , Alelos , Anemia Falciforme/diagnóstico , Anemia Falciforme/genética , Pré-Escolar , Hemoglobina Falciforme/análise , Humanos , Recém-Nascido , Técnicas de Amplificação de Ácido Nucleico , Sistemas Automatizados de Assistência Junto ao Leito , Sensibilidade e EspecificidadeRESUMO
Management of fever is a key element of care for children with sickle cell disease (SCD). There exist few studies of current practices in managing fevers in SCD in low- and middle-income countries (LMICs) and malaria-endemic regions where SCD is prevalent. We surveyed medical providers in these settings to characterize current practices in infection prevention and fever management for children with SCD. We found wide variation in use of newborn screening for early diagnosis and infection prevention, pneumococcal vaccination, use of antibiotics and antimalarials, and route of antibiotic administration. Counter to established guidelines, 78% (95% CI: 59-100%) of respondents would consider using oral antibiotics for a febrile child with SCD. Only 17% (95% CI: 0-37%) would administer antibiotics to a well-appearing child with a positive malaria test. Availability of blood cultures did not affect duration of antibiotic course. Further study and standardization of fever management in SCD in LMICs are urgently needed.
Assuntos
Anemia Falciforme/complicações , Anemia Falciforme/diagnóstico , Países em Desenvolvimento , Febre/diagnóstico , Febre/tratamento farmacológico , Anemia Falciforme/terapia , Antibacterianos/administração & dosagem , Antibacterianos/uso terapêutico , Antimaláricos/uso terapêutico , Humanos , Injeções Intravenosas , Malária/tratamento farmacológico , Guias de Prática Clínica como AssuntoAssuntos
Anemia Falciforme , Cuidadores , África , Criança , Feminino , Humanos , Mães , Medição de RiscoRESUMO
This study compared outcomes following total (TS) or partial splenectomy (PS) among patients with hereditary spherocytosis. Seventy-nine patients (TS = 33, PS = 46) were identified. The follow-up period was longer after PS (59.6 vs. 24.9 months, p < .001). Long-term adverse events occurred more frequently following PS (50% vs. 29%, p = .001). Anemia, jaundice, and fatigue recurred in six patients with PS, leading to five completion splenectomies. Hemoglobin was not different between PS and TS by 5 years post-procedure (12.3 vs. 13.4 g/dL, p = .25). Both PS and TS ameliorate symptoms and improve hematologic parameters. The rate of secondary surgery following PS should be considered when planning the initial surgical procedure.