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Safety of Ferric Carboxymaltose in Children: Report of a Case Series from Greece and Review of the Literature
Panagopoulou P, Alexiadou S, Ntoumpara M, Papazoglou A, Makis A, Tragiannidis A, Fotoulaki M, Mantadakis E
Paediatric drugs. 2022
Abstract
BACKGROUND Parenteral iron is generally considered safe in adults, and severe adverse events are extremely rare. Ferric carboxymaltose (FCM), a third-generation parenteral iron product, is not licensed for pediatric use. OBJECTIVE The aim of this study was to present our data on the safety of FCM in children with iron deficiency (ID) and/or iron deficiency anemia (IDA) and to investigate through a systematic literature review articles reporting on the safety of FCM use in children with ID/IDA. PATIENTS AND METHODS Safety data regarding children treated with FCM for ID/IDA from four pediatric departments in Greece over a 26-month period are presented. Additionally, a literature search was performed in PubMed, Scopus, and Google Scholar on December 4, 2021 for articles reporting on the use of FCM in children with ID/IDA. Review articles, guidelines, case reports/case series, and reports on the use of FCM for conditions other than ID/IDA were excluded. Identified articles were screened for all reported adverse events (AE) that were graded according to the Common Terminology Criteria for Adverse Events, version 5.0. RESULTS In our cohort, 37 children with ID/IDA received 41 FCM infusions. All infusions were tolerated well. In addition, 11 articles reporting 1231 infusions of FCM in 866 children were identified in the literature. Among them, 52 (6%) children developed AE that were graded as mild or moderate (grades I-III). CONCLUSIONS Our patient cohort and this literature review provide further evidence for the good safety profile of FCM in children, although well-designed prospective clinical trials with appropriate safety endpoints are still required.
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A Systematic review on diagnostic methods of red cell membrane disorders in Asia
Silva R, Amarasinghe D, Perera S, Premawardhena A
International journal of laboratory hematology. 2022
Abstract
Membranopathies are a group of inherited blood disorders where the diagnosis could form a challenge due to phenotype-genotype heterogeneity. In this review, the usage and limitations of diagnostic methods for membranopathies in Asian countries were evaluated. A systematic review was done using articles from PubMed, Google Scholar, and EBSCO from 2000 to 2020. Thirty-six studies conducted in seven Asian countries had used different diagnostic methods to confirm membranopathies. In 58.3% of studies, full blood count (FBC), reticulocyte count, and peripheral blood smear (PBS) were used in preliminary diagnosis. The combination of the above three with osmotic fragility (OF) test was used in 38.8%. The flowcytometric osmotic fragility (FC-OF) test was used in 27.7% where it showed high sensitivity (92%-100%) and specificity (96%-98%). The eosin-5-maleimide (EMA) assay was used in 68.1% with high sensitivity (95%-100%) and specificity (93%-99.6%). About 36.1% of studies had used sodium dodecyl sulfate-polyacrylamide gel electrophoresis (SDS-PAGE) as a further diagnostic method to detect defective proteins. Genetic analysis to identify mutations was done using Sanger sequencing, next-generation sequencing (NGS), and whole-exome sequencing (WES) in 33.3%, 22.2%, and 13.8% of studies, respectively. The diagnostic yield of NGS ranged from 63% to 100%. Proteomics was used in 5.5% of studies to support the diagnosis of membranopathies. A single method could not diagnose all membranopathies. Next-generation sequencing, Sanger sequencing, and proteomics will supplement the well-established screening and confirmatory methods, but not replace them in hereditary hemolytic anemia assessment.
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Efficacy and safety of intravenous iron with different frequencies for renal anaemia: A systematic review and meta-analysis
Chen T, Deng Y, Gong R
Journal of clinical pharmacy and therapeutics. 2022
Abstract
WHAT IS KNOWN AND OBJECTIVE To evaluate the efficacy and safety of intravenous iron supplementation in patients with renal anaemia. METHODS We searched the PubMed, Embase, Cochrane Library, and Web of Science from their inception until 17 September 2021, for randomized controlled trials (RCTs) to evaluate the efficacy and safety of intravenous iron at different frequencies. The observed efficacy indicators included transfer saturation (TSAT), serum ferritin (SF) and haemoglobin (HGB). Outcomes of interest included allergies, infections, all-cause mortality and cardiovascular events. RESULTS AND DISCUSSION Of the 751 eligible studies, 7 RCTs met the inclusion criteria. The RCTs showed that there were no significant differences between the low-frequency high-dose group (1-2 doses, >200 mg/dose) and the high-frequency low-dose group (4-5 doses, ≤200 mg/dose) in the increase in TSAT (WMD = 1.90; 95% CI = -2.04 to 5.84; I(2) = 0%), SF (WMD = 15.70; 95% CI = -32.20 to 70.61; I(2) = 0%) and HGB (WMD = -0.00; 95% CI = -0.43 to 0.42; I(2) = 0%). There was also no significant difference in the occurrence of outcome events, including allergies (RR = 1.84; 95% CI = 0.95 to 3.57; I(2) = 45%), infections (RR = 0.61; 95% CI = 0.20-1.86; I(2) = 0%), cardiovascular events (RR = 0.88; 95% CI = 0.67-1.15; I(2) = 48%) and all-cause mortality (RR = 0.74; 95% CI = 0.40-1.35; I(2) = 0%). WHAT IS NEW AND CONCLUSION Frequencies of intravenous iron supplementation with similar doses share similar safety and efficacy in patients with renal anaemia. However, a single dose or two doses of intravenous iron are more cost-effective and patient friendly. These findings may provide evidence for the clinical application of intravenous iron supplementation for patients with renal anaemia.
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Role of gene therapy in Fanconi anemia: A systematic and literature review with future directions
Shafqat S, Tariq E, Parnes AD, Dasouki MJ, Ahmed SO, Hashmi SK
Hematology/oncology and stem cell therapy. 2021
Abstract
Gene therapy (GT) has been reported to improve bone marrow function in individuals with Fanconi anemia (FA); however, its clinical application is still in the initial stages. We conducted this systematic review, following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines, to assess the long-term safety and clinical outcomes of GT in FA patients. Electronic searches from PubMed, Web of Science, Cochrane Library, and Google Scholar were conducted and full texts of articles meeting our inclusion criteria were reviewed. Three clinical trials were included, with a total of nine patients and mean age of 10.7 ± 5.7 years. All patients had lentiviral-mediated GT. A 1-year follow-up showed stabilization in blood lineages, without any serious adverse effects from GT. A metaregression analysis could not be conducted, as very little long-term follow-up data of patients was observed, and the median survival rate could not be calculated. Thus, we can conclude that GT seems to be a safe procedure in FA; however, further research needs to be conducted on the longitudinal clinical effects of GT in FA, for a better insight into its potential to become a standard form of treatment.
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Disease-modifying treatments for primary autoimmune haemolytic anaemia
Liu AP, Cheuk DK
The Cochrane database of systematic reviews. 2021;3:Cd012493
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Editor's Choice
Abstract
BACKGROUND Primary autoimmune haemolytic anaemia (AIHA) is an autoantibody mediated condition characterised by a variable disease course. A myriad of immunomodulatory agents have been employed but there is a paucity of evidence to support their use or compare their effectiveness. OBJECTIVES To determine the effects of various disease-modifying treatment modalities in people with AHIHA. SEARCH METHODS We searched MEDLINE (Ovid) (1946 to 2021), Embase (Ovid) (1974 to 2021), Latin American and Caribbean Health Sciences Literature (LILACS) (1982 to 2021), and the Cochrane Library (CENTRAL). Clinical trial registries and relevant conference proceedings were also reviewed. Records were included as of 7 March 2021. We did not impose any language restrictions. SELECTION CRITERIA Randomised controlled trials (RCTs) comparing immunosuppressive or immunomodulatory treatments against no treatment, placebo, or another immunosuppressive or immunomodulatory treatment, for people of all age with idiopathic AIHA. DATA COLLECTION AND ANALYSIS We used standard methodological procedures expected by Cochrane. The prioritised pre-defined outcomes included complete haematological response at 12 months, frequency of adverse events at two, six and 12 months, partial haematological response at 12 months, overall survival at six and 12 months, relapse-free survival (RFS) at six and 12 months, red blood cel (RBC) transfusion requirement after treatment at 12 months, and quality of life (QOL) as measured by validated instruments at 12 months. Based on data availability, we were only able to perform meta-analysis on frequency of complete haematological response. MAIN RESULTS Two trials were included, enrolling a total of 104 adult participants (96 randomised) with warm AIHA in the setting of tertiary referral centres, both comparing the effectiveness between rituximab (375 mg/m(2) weekly for four weeks, or 1000 mg for two doses two weeks apart) plus glucocorticoid (prednisolone 1.5 or 1mg/kg/day with taper) and glucocorticoid monotherapy. The average age of participants in the two trials were 67 and 71, respectively. One of the included studies had good methodological quality with low risk of bias, whereas the other study had high risk of performance and detection bias due to lack of blinding. Compared with glucocorticoid alone, adding rituximab may result in a large increase of complete response at 12 months (n = 96, risk ratio (RR) 2.13, 95% confidence interval (CI) 1.34 to 3.40, GRADE low-certainty evidence). Rates of adverse effects at prespecified time-points were not reported. Limited data on partial haematological response were reported. The evidence is very uncertain about the effect of adding rituximab to glucocorticoids on partial haematological response at 12 months (n = 32; study = 1; RR 3.00, 95% CI 0.13 to 68.57; GRADE very low-certainty evidence). RBC transfusion need at 12 months was reported in one study, with four participants (mean number of packed red cell units 4.0 ± 2.82) from the rituximab group and five participants from the placebo (corticosteroid only) (mean number of packed red cell units 5.6 ± 4.15) group requiring transfusion, indicating very uncertain evidence about the effect of adding rituximab to glucocorticoids (n = 32, RR 0.80, 95% CI 0.26 to 2.45, GRADE very low-certainty evidence). The other study did not report transfusion requirement at prespecified time points but reported no difference in transfusion requirement between the two groups when comparing responders from enrolment to end of response or to the end of study follow-up (34 units versus 30 units, median [range]: 0 [1 to 6] versus 0 [1 to 5], P = 0·81). Overall survival and RFS rates at prespecified time-points were not explicitly reported in either study. Data on QOL were not available. AUTHORS' CONCLUSIONS Available literature on the effectiveness of immunomodulatory therapy for primary AIHA is restricted to comparison between rituximab plus glucocorticoid and glucocorticoid alone, in patients with newly diagnosed warm AIHA, calling for need for additional studies. The current result suggests that combinatory therapy with rituximab and glucocorticoid may increase the rate of complete haematological response over glucocorticoid monotherapy.
PICO Summary
Population
Primary autoimmune haemolytic anaemia (AIHA) patients (2 studies, n= 104).
Intervention
Rituximab plus glucocorticoid.
Comparison
Glucocorticoid.
Outcome
Compared with glucocorticoid alone, adding rituximab may result in a large increase of complete response at 12 months. Rates of adverse effects at pre-specified time-points were not reported. The evidence is very uncertain about the effect of adding rituximab to glucocorticoids on partial haematological response at 12 months. Red blood cell transfusion need at 12 months was reported in one study. The other study did not report transfusion requirement at pre-specified time points but reported no difference in transfusion requirement between the two groups.
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Serum or plasma ferritin concentration as an index of iron deficiency and overload
Garcia-Casal MN, Pasricha SR, Martinez RX, Lopez-Perez L, Peña-Rosas JP
The Cochrane database of systematic reviews. 2021;5(5):Cd011817
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Editor's Choice
Abstract
BACKGROUND Reference standard indices of iron deficiency and iron overload are generally invasive, expensive, and can be unpleasant or occasionally risky. Ferritin is an iron storage protein and its concentration in the plasma or serum reflects iron stores; low ferritin indicates iron deficiency, while elevated ferritin reflects risk of iron overload. However, ferritin is also an acute-phase protein and its levels are elevated in inflammation and infection. The use of ferritin as a diagnostic test of iron deficiency and overload is a common clinical practice. OBJECTIVES To determine the diagnostic accuracy of ferritin concentrations (serum or plasma) for detecting iron deficiency and risk of iron overload in primary and secondary iron-loading syndromes. SEARCH METHODS We searched the following databases (10 June 2020): DARE (Cochrane Library) Issue 2 of 4 2015, HTA (Cochrane Library) Issue 4 of 4 2016, CENTRAL (Cochrane Library) Issue 6 of 12 2020, MEDLINE (OVID) 1946 to 9 June 2020, Embase (OVID) 1947 to week 23 2020, CINAHL (Ebsco) 1982 to June 2020, Web of Science (ISI) SCI, SSCI, CPCI-exp & CPCI-SSH to June 2020, POPLINE 16/8/18, Open Grey (10/6/20), TRoPHI (10/6/20), Bibliomap (10/6/20), IBECS (10/6/20), SCIELO (10/6/20), Global Index Medicus (10/6/20) AIM, IMSEAR, WPRIM, IMEMR, LILACS (10/6/20), PAHO (10/6/20), WHOLIS 10/6/20, IndMED (16/8/18) and Native Health Research Database (10/6/20). We also searched two trials registers and contacted relevant organisations for unpublished studies. SELECTION CRITERIA We included all study designs seeking to evaluate serum or plasma ferritin concentrations measured by any current or previously available quantitative assay as an index of iron status in individuals of any age, sex, clinical and physiological status from any country. DATA COLLECTION AND ANALYSIS We followed standard Cochrane methods. We designed the data extraction form to record results for ferritin concentration as the index test, and bone marrow iron content for iron deficiency and liver iron content for iron overload as the reference standards. Two other authors further extracted and validated the number of true positive, true negative, false positive, false negative cases, and extracted or derived the sensitivity, specificity, positive and negative predictive values for each threshold presented for iron deficiency and iron overload in included studies. We assessed risk of bias and applicability using the Quality Assessment of Diagnostic Accuracy Studies (QUADAS)-2 tool. We used GRADE assessment to enable the quality of evidence and hence strength of evidence for our conclusions. MAIN RESULTS Our search was conducted initially in 2014 and updated in 2017, 2018 and 2020 (10 June). We identified 21,217 records and screened 14,244 records after duplicates were removed. We assessed 316 records in full text. We excluded 190 studies (193 records) with reasons and included 108 studies (111 records) in the qualitative and quantitative analysis. There were 11 studies (12 records) that we screened from the last search update and appeared eligible for a future analysis. We decided to enter these as awaiting classification. We stratified the analysis first by participant clinical status: apparently healthy and non-healthy populations. We then stratified by age and pregnancy status as: infants and children, adolescents, pregnant women, and adults. Iron deficiency We included 72 studies (75 records) involving 6059 participants. Apparently healthy populations Five studies screened for iron deficiency in people without apparent illness. In the general adult population, three studies reported sensitivities of 63% to 100% at the optimum cutoff for ferritin, with corresponding specificities of 92% to 98%, but the ferritin cutoffs varied between studies. One study in healthy children reported a sensitivity of 74% and a specificity of 77%. One study in pregnant women reported a sensitivity of 88% and a specificity of 100%. Overall confidence in these estimates was very low because of potential bias, indirectness, and sparse and heterogenous evidence. No studies screened for iron overload in apparently healthy people. People presenting for medical care There were 63 studies among adults presenting for medical care (5042 participants). For a sample of 1000 subjects with a 35% prevalence of iron deficiency (of the included studies in this category) and supposing a 85% specificity, there would be 315 iron-deficient subjects correctly classified as having iron deficiency and 35 iron-deficient subjects incorrectly classified as not having iron deficiency, leading to a 90% sensitivity. Thresholds proposed by the authors of the included studies ranged between 12 to 200 µg/L. The estimated diagnostic odds ratio was 50. Among non-healthy adults using a fixed threshold of 30 μg/L (nine studies, 512 participants, low-certainty evidence), the pooled estimate for sensitivity was 79% with a 95% confidence interval of (58%, 91%) and specificity of 98%, with a 95% confidence interval of (91%, 100%). The estimated diagnostic odds ratio was 140, a relatively highly informative test. Iron overload We included 36 studies (36 records) involving 1927 participants. All studies concerned non-healthy populations. There were no studies targeting either infants, children, or pregnant women. Among all populations (one threshold for males and females; 36 studies, 1927 participants, very low-certainty evidence): for a sample of 1000 subjects with a 42% prevalence of iron overload (of the included studies in this category) and supposing a 65% specificity, there would be 332 iron-overloaded subjects correctly classified as having iron overload and 85 iron-overloaded subjects incorrectly classified as not having iron overload, leading to a 80% sensitivity. The estimated diagnostic odds ratio was 8. AUTHORS' CONCLUSIONS At a threshold of 30 micrograms/L, there is low-certainty evidence that blood ferritin concentration is reasonably sensitive and a very specific test for iron deficiency in people presenting for medical care. There is very low certainty that high concentrations of ferritin provide a sensitive test for iron overload in people where this condition is suspected. There is insufficient evidence to know whether ferritin concentration performs similarly when screening asymptomatic people for iron deficiency or overload.
PICO Summary
Population
People of any age, sex, clinical or psychological condition having their iron status analysed as part of a clinical study (n= 6,059, 72 studies).
Intervention
Different methods of assessing iron deficiency and iron overload in people without apparent illness.
Comparison
Different methods of assessing iron deficiency and iron overload in people presenting for medical care.
Outcome
Apparently healthy populations: In the general adult population, three studies reported sensitivities of 63% to 100% at the optimum cut-off for ferritin, with corresponding specificities of 92% to 98%, but the ferritin cut-offs varied between studies. One study in healthy children reported a sensitivity of 74% and a specificity of 77%. One study in pregnant women reported a sensitivity of 88% and a specificity of 100%. Overall confidence in these estimates was very low because of potential bias, indirectness, and sparse and heterogenous evidence. No studies screened for iron overload in apparently healthy people. People presenting for medical care: There were 63 studies among adults presenting for medical care (5,042 participants). For a sample of 1,000 subjects with a 35% prevalence of iron deficiency and supposing a 85% specificity, there would be 315 iron-deficient subjects correctly classified as having iron deficiency and 35 iron-deficient subjects incorrectly classified as not having iron deficiency, leading to a 90% sensitivity. Thresholds proposed by the authors of the included studies ranged between 12 to 200 µg/L. The estimated diagnostic odds ratio was 50. Among non-healthy adults using a fixed threshold of 30 μg/L (nine studies, 512 participants, low-certainty evidence), the pooled estimate for sensitivity was 79% with a 95% confidence interval of (58%, 91%) and specificity of 98%, with a 95% confidence interval of (91%, 100%). The estimated diagnostic odds ratio was 140, a relatively highly informative test. Iron overload: We included 36 studies (36 records) involving 1,927 participants. All studies concerned non-healthy populations. There were no studies targeting either infants, children, or pregnant women. Among all populations (one threshold for males and females; 36 studies, 1,927 participants, very low-certainty evidence): for a sample of 1000 subjects with a 42% prevalence of iron overload (of the included studies in this category) and supposing a 65% specificity, there would be 332 iron-overloaded subjects correctly classified as having iron overload and 85 iron-overloaded subjects incorrectly classified as not having iron overload, leading to a 80% sensitivity. The estimated diagnostic odds ratio was 8.
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Role of Erythropoiesis Stimulating Agents in the Treatment of Anemia: a Literature Review
Kidanewold A, Woldu B, Enawgaw B
Clinical laboratory. 2021;67(4)
Abstract
BACKGROUND Erythropoiesis stimulating agents are exogenous erythropoietin medications that are used to stimulate the bone marrow red blood cells' production for the management of anemia of chronic kidney disease, some anticancer drugs, myelodysplastic syndrome, and others. Currently, there are different erythropoiesis stimulating agents accessible in the market. The objective of this narrative literature review is to summarize the role of some erythropoiesis stimulating agents in the treatment of anemia. METHODS The following method was used to prepare this narrative literature review. The comprehensive computerized search of literatures was carried out using PubMed, Cochrane library, Google scholar, and Science direct. Keywords such as recombinant human erythropoietin, epoetin, darbepoetin, continuous erythropoietin receptor agonist, pegzyrepoetin alfa, erythropoiesis stimulating agents in combination with anemia/anaemia were used. The pertinent original and review full articles which are written in the English language were included in this narrative review. RESULTS From the discussions of the literature, erythropoiesis stimulating agents that are produced by different biosimilar manufacturers have different clinical characteristics and stabilities as a result of their chemical modifications. The chemical modifications of erythropoiesis stimulating agents like glycosylation and polyethylene glycosylation determine the half-life, affinity to erythropoietin receptor, and immune response of the agents. Erythro-poiesis stimulating agents are categorized as short-acting and long-acting agents due to their chemical structures that influence the clinical efficacy and safety of the agents. CONCLUSIONS The effectiveness of the agents is different in different patients depending on the individual characteristics and etiologies of anemia. The agents not only have the benefits but also, they have the risks for the patients. Hence, the risks and benefits of erythropoiesis stimulating agents must be given special consideration in the managements of anemia to get maximum efficacy for anemic patients. The treatment is dependent on hemoglobin levels of individual patients. The physician must follow the patients during and after therapy using erythropoiesis stimulating agents.
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Comparative Risk of Hypophosphatemia Following the Administration of Intravenous Iron Formulations: A Network Meta-Analysis
Bellos I, Frountzas M, Pergialiotis V
Transfusion medicine reviews. 2020
Abstract
Intravenous iron therapy is increasingly used in patients with iron deficiency anemia, although concerns of hypophosphatemia have been recently raised. The aim of this study was to evaluate different intravenous iron formulations for the risk of hypophosphatemia. Medline, Scopus, Cochrane Central Register of Controlled Trials, Web of Science, Clinicaltrials.gov, and Google Scholar databases were systematically searched to 20 March 2020. All randomized controlled trials reporting the incidence of hypophosphatemia among adult patients treated with any intravenous iron preparation were included. Pool estimates were obtained by applying an arm-based Bayesian network meta-analysis model. Eight randomized controlled trials were included, comprising 5989 patients. Ferric carboxymaltose was associated with significantly higher incidence of hypophosphatemia compared to iron isomaltoside (risk ratio [RR]: 7.90, 95% confidence interval [CI]: 2.10-28.0), iron sucrose (RR: 9.40, 95% CI: 2.30-33.0), iron dextran (RR: 6.60, 95% CI: 1.91-220.0), and ferumoxytol (RR: 24.0, 95% CI: 2.50-220.0). Therefore, ferric carboxymaltose ranked as the worst treatment presenting the highest surface under the cumulative ranking curve (99.1%). No significant differences were estimated for the comparisons among iron isomaltoside, iron sucrose, iron dextran, and ferumoxytol. In conclusion, it is suggested that the occurrence of hypophosphatemia is common after the administration of intravenous ferric carboxymaltose. Further research is needed in large-scale randomized controlled trials to determine the risk of symptomatic and persistent hypophosphatemia as well as to elucidate the exact pathophysiology of the observed association.
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Systematic review of guidelines for the diagnosis and treatment of iron deficiency anemia using intravenous (iv) iron across multiple indications
Numan S, Kaluza K
Current medical research and opinion. 2020;:1
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Editor's Choice
Abstract
Objective: To explore current recommendations for intravenous (IV) iron use in clinical guidelines for iron deficiency anemia (IDA) across different therapeutic areas and identify recommendations, if any, for the treatment of IDA.Methods: A literature search was conducted in Medline, EMBASE, BIOSIS, Cochrane Collaboration, and on websites of relevant professional associations. Searches were limited to English publications. 1,292 citations were identified, 219 papers were assessed, and 35 guidelines were identified for inclusion.Results: The guidelines covered a variety of geographies: United States (US; n = 10); Europe (n = 11); 'Rest-of-World' (n = 9); and 'Other' organizations (n = 5). These covered a variety of specialties. Guidelines defined iron deficiency and IDA generally by serum ferritin and transferrin saturation levels. One-fifth of the reviewed guidelines (7 of 35) included no mention or recommendation regarding parenteral iron's utility in the management of IDA. Fifteen guidelines recommended using parenteral iron in the management of IDA. Fewer US guidelines included recommendations around IV iron than in Europe or the rest of the world. Approximately 60% of the guidelines have not been updated in ≥5 years and consequently do not reflect current evidence on the safety and efficacy of IV iron.Conclusions: While national and international guidelines for management of IDA exist, many are outdated and do not reflect current evidence including, but not limited to, parenteral iron use. Urgent consideration should be given to updating and clarifying management guidelines for IDA using the latest treatment modalities and options, particularly in the US.
PICO Summary
Population
Iron deficiency anaemia (IDA) patients (219 papers and 35 guidelines).
Intervention
Systematic review of guidelines for the diagnosis and treatment of iron deficiency anemia using intravenous iron (IV).
Comparison
Outcome
The guidelines covered: United States (n = 10), Europe (n = 11), 'Rest-of-World' (n = 9), and 'Other' organizations (n = 5): and a variety of specialties. Guidelines defined iron deficiency and IDA generally by serum ferritin and transferrin saturation levels. Fifteen guidelines recommended using parenteral iron in the management of IDA. Fewer US guidelines included recommendations around IV iron than in Europe or the rest of the world. Approximately 60% of the guidelines have not been updated in ≥5 years.
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10.
The efficacy of voxelotor, 900 mg in patients with sickle cell anemia: a meta-analysis of the randomized controlled trials
Tanriverdi LH, Sarici A, Erkurt MA, Parlakpinar H
International journal of clinical practice. 2020;:e13967
Abstract
Sickle cell anemia (SCA) is an autosomal recessive disease caused by a mutation in the gene that codes the β-globin chain of hemoglobin. The abnormal hemoglobin (hemoglobin S) can polymerize when deoxygenated, changing the physical properties of the red cells (which deform to a sickle shape, hence the name) and damages cell membranes.(1) Sickle cell anemia can lead to a variety of signs and symptoms namely vaso-occlusive attacks (presenting as pain, tissue hypoxia, necrosis, and organ damage), acute chest syndrome, hemolytic crisis, and primary and secondary stroke.