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1.
Am J Hematol ; 95(6): 643-651, 2020 06.
Artigo em Inglês | MEDLINE | ID: mdl-32129511

RESUMO

Romiplostim self-administration by patients or caregivers may offer time/cost savings to healthcare professionals (HCPs) and convenience for patients who avoid weekly clinic visits. We performed an integrated analysis of five clinical trials to evaluate the efficacy and safety of romiplostim self-administration. Data were analyzed from adults with immune thrombocytopenia (ITP) who received weekly romiplostim via self-administration or from an HCP. Patients who achieved a stable romiplostim dose for ≥3 weeks (HCP group ≥5 weeks to provide an appropriate index date to enable comparisons with the self-administration group) with platelet counts ≥50 × 109 /L were eligible. In the self-administration (n = 621) vs HCP (n = 133) groups, respectively, median age was 53 vs 58 years, median time since primary ITP diagnosis was 3.7 vs 2.5 years, and median baseline platelet count at ITP diagnosis was 19.0 vs 20.0 × 109 /L. In the self-administration and HCP-dosed groups, median romiplostim treatment duration was 89 vs 52 weeks and median total number of doses was 81 vs 50, respectively. In the self-administration and HCP groups, respectively: 95.0% and 100.0% of patients achieved ≥1 platelet response (defined as weekly platelet count ≥50 × 109 /L without rescue medication in previous 4 weeks); the median percentage of weeks with a response was 94.5% and 95.9%; and rescue medication was used in 36.7% and 39.8% of patients. Self-administration did not adversely affect safety; duration-adjusted rates for all treatment-emergent adverse events (TEAEs) and bleeding TEAEs were numerically lower with self-administration. Romiplostim self-administration appears effective and well tolerated in eligible patients with ITP.


Assuntos
Bases de Dados Factuais , Púrpura Trombocitopênica Idiopática/tratamento farmacológico , Receptores Fc/administração & dosagem , Proteínas Recombinantes de Fusão/administração & dosagem , Trombopoetina/administração & dosagem , Adolescente , Adulto , Idoso , Idoso de 80 Anos ou mais , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Contagem de Plaquetas , Púrpura Trombocitopênica Idiopática/sangue , Proteínas Recombinantes de Fusão/efeitos adversos , Autoadministração , Trombopoetina/efeitos adversos
2.
Medicine (Baltimore) ; 99(7): e19096, 2020 Feb.
Artigo em Inglês | MEDLINE | ID: mdl-32049816

RESUMO

INTRODUCTION: The assessment of bone marrow thrombopoietic activity in patients with thrombocytopenia is necessary to achieve an accurate diagnosis and administer effective treatment. We evaluated the discriminatory power of the immature platelet fraction (IPF) in differentiating hyperdestructive/consumptive thrombocytopenia from hypoproductive thrombocytopenia and its potential use as a predictive marker for platelet recovery. METHODS: In this observational study, platelet indices, including IPF, were measured in 105 healthy individuals, 27 patients with hyperdestructive/consumptive thrombocytopenia (all with immune thrombocytopenic purpura [ITP]), and 35 patients with hypoproductive thrombocytopenia (5 with aplastic anemia and 30 with cancer who were undergoing chemotherapy) using a Sysmex XN-3000 hematology analyzer. RESULTS: The platelet distribution width, mean platelet volume, platelet large cell ratio, IPF, and absolute immature platelet count (AIPC) were significantly higher in the hyperdestructive/consumptive thrombocytopenia group than in the hypoproductive thrombocytopenia group (P < .001). The IPF showed the highest difference between the two patient groups (200%). Receiver operating characteristics analysis that showed the IPF had the largest area under the curve among all the platelet indices analyzed; its cut-off value was 2.3%. The IPF decreased 3 to 4 days in advance of platelet count elevation in patients with ITP, whereas the delta AIPC increased 3 days in advance. Furthermore, the IPF and delta AIPC increased 5.5 days and 8.5 days, respectively, before platelet counts increased up to 130.0 × 10/L in cancer patients receiving chemotherapy. CONCLUSION: These data demonstrated that the IPF and delta AIPC are both excellent indicators of the etiology of thrombocytopenia and predictive markers for platelet recovery.


Assuntos
Anemia Aplástica/diagnóstico , Volume Plaquetário Médio , Contagem de Plaquetas , Púrpura Trombocitopênica Idiopática/diagnóstico , Adulto , Idoso , Anemia Aplástica/sangue , Anemia Aplástica/complicações , Biomarcadores/sangue , Estudos de Casos e Controles , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Neoplasias/complicações , Valor Preditivo dos Testes , Púrpura Trombocitopênica Idiopática/sangue , Curva ROC
3.
Medicine (Baltimore) ; 99(1): e18624, 2020 Jan.
Artigo em Inglês | MEDLINE | ID: mdl-31895820

RESUMO

The purpose of this study was to evaluate neutropenia following intravenous immunoglobulin (IVIG) therapy in adults with immune thrombocytopenic purpura (ITP).Our analysis included 88 patients with ITP, who received IVIG from January 2006 to March 2016, at Pusan National University Hospital in Korea. Their white blood cell (WBC) count and absolute neutrophil count (ANC) before and after IVIG treatment were analyzed.Of 88 patients, 24 patients (27.3%) were male, and 64 patients (72.7%) were female. Neutropenia developed in 8 patients (18.7%) after IVIG treatment. In patients with a decrease in WBC count and ANC compared to baseline, median WBC count decreased from 6280/µL to 4530/µL after IVIG therapy, and median ANC decreased from 3840/µL to 2840/µL after IVIG therapy. The neutropenia induced by IVIG had resolved spontaneously after several days, and the mean recovery time was 8.72 days after the completion of the IVIG treatment. During the neutropenic episodes, only one patient developed neutropenic fever, which subsided soon without any treatment.The results of this study suggest that IVIG may cause neutropenia commonly in adults with ITP, and it seems to be transient and self-limited. This study is meaningful as the first report that not only pediatric ITP patients may develop neutropenia post IVIG administration, but also adult patients suffering ITP.


Assuntos
Imunoglobulinas Intravenosas/efeitos adversos , Neutropenia/etiologia , Púrpura Trombocitopênica Idiopática/terapia , Adolescente , Adulto , Idoso , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Púrpura Trombocitopênica Idiopática/sangue , Púrpura Trombocitopênica Idiopática/imunologia , Estudos Retrospectivos , Adulto Jovem
4.
Am J Hematol ; 95(2): 178-187, 2020 02.
Artigo em Inglês | MEDLINE | ID: mdl-31821591

RESUMO

Primary immune thrombocytopenia (ITP) is an acquired autoimmune bleeding disorder, characterized by a low platelet count (<100 × 109 /L) in the absence of other causes associated with thrombocytopenia. In most patients, IgG autoantibodies directed against platelet receptors can be detected. They accelerate platelet clearance and destruction, inhibit platelet production, and impair platelet function, resulting in increased risk of bleeding and impaired quality of life. Efgartigimod is a human IgG1 antibody Fc-fragment, a natural ligand of the neonatal Fc receptor (FcRn), engineered for increased affinity to FcRn, while preserving its characteristic pH-dependent binding. Efgartigimod blocks FcRn, preventing IgG recycling, and causing targeted IgG degradation. In this Phase 2 study, 38 patients were randomized 1:1:1 to receive four weekly intravenous infusions of either placebo (N = 12) or efgartigimod at a dose of 5 mg/kg (N = 13) or 10 mg/kg (N = 13). This short treatment cycle of efgartigimod in patients with ITP, predominantly refractory to previous lines of therapy, was shown to be well tolerated, and demonstrated a favorable safety profile consistent with Phase 1 data. Efgartigimod induced a rapid reduction of total IgG levels (up to 63.7% mean change from baseline), which was associated with clinically relevant increases in platelet counts (46% patients on efgartigimod vs 25% on placebo achieved a platelet count of ≥50 × 109 /L on at least two occasions, and 38% vs 0% achieved ≥50 × 109 /L for at least 10 cumulative days), and a reduced proportion of patients with bleeding. Taken together, these data warrant further evaluation of FcRn antagonism as a novel therapeutic approach in ITP.


Assuntos
Fragmentos Fc das Imunoglobulinas/uso terapêutico , Imunoglobulina G/uso terapêutico , Púrpura Trombocitopênica Idiopática , Receptores Fc/antagonistas & inibidores , Adulto , Idoso , Método Duplo-Cego , Feminino , Seguimentos , Antígenos de Histocompatibilidade Classe I/sangue , Humanos , Masculino , Pessoa de Meia-Idade , Contagem de Plaquetas , Púrpura Trombocitopênica Idiopática/sangue , Púrpura Trombocitopênica Idiopática/tratamento farmacológico , Receptores Fc/sangue
6.
BMC Cancer ; 19(1): 1058, 2019 Nov 06.
Artigo em Inglês | MEDLINE | ID: mdl-31694583

RESUMO

BACKGROUND: This report illustrates the importance of a detailed history and physical exam and careful analysis of hematologic parameters when diagnosing ITP. This case demonstrates that even with subtle deviations from typical ITP findings one must promptly reevaluate the diagnosis. This case also highlights the importance of peripheral smear review by an expert in pediatric hematopathology. CASE PRESENTATION: A previously healthy 10 year-old Asian boy presented with 2 months of easy bruising. Review of systems was negative for any constitutional symptoms. On examination, he appeared well but had numerous large ecchymoses. He had no appreciable lymphadenopathy or splenomegaly. The liver was palpable 1.5 cm below the costal margin. A complete blood count (CBC) showed: platelets = 17 × 109/L, hemoglobin = 128 g/L, white blood cell count = 5.43 × 109/L, and neutrophils = 1.63 × 109/L. A blood smear was reported as normal. Urate was 370 umol/L and lactate dehydrogenase (LDH) was 803 U/L. The child was admitted with a presumptive diagnosis of immune thrombocytopenic purpura (ITP) and treated with intravenous immunoglobulin. The following day, the blood smear was reviewed by a hematopathologist who identified blasts. A bone marrow aspiration (BMA) confirmed the diagnosis of precursor B-cell acute lymphoblastic leukemia. CONCLUSION: In children presenting with suspected ITP, leukemia should always be considered. A BMA was historically performed on all patients with presumed ITP to rule out leukemia. In 2011, the American Society of Hematology (ASH) stopped recommending routine BMA in patients suspected of having ITP. ASH advises in cases with unusual findings on history, physical examination or CBC, it is reasonable to perform a BMA. Our patient had mild hepatomegaly, which may have qualified him for a BMA. He also had an elevated LDH and urate, which are not listed as criteria for BMA by ASH but were considered atypical for ITP by the clinical team. A literature search did not reveal any primary data assessing these markers. While corticosteroids are a first line treatment in ITP, they must be reserved for when clinicians are confident that the patient does not have leukemia. Steroid administration prior to diagnosing leukemia results in delayed diagnosis and may increase the risk of complications and decrease survival.


Assuntos
Imunoglobulinas Intravenosas/uso terapêutico , Fígado/patologia , Leucemia-Linfoma Linfoblástico de Células Precursoras B/diagnóstico , Púrpura Trombocitopênica Idiopática/diagnóstico , Púrpura Trombocitopênica Idiopática/tratamento farmacológico , Exame de Medula Óssea , Criança , Diagnóstico Diferencial , Humanos , Imunoglobulinas Intravenosas/administração & dosagem , Masculino , Leucemia-Linfoma Linfoblástico de Células Precursoras B/sangue , Púrpura Trombocitopênica Idiopática/sangue
7.
Med Sci Monit ; 25: 8683-8693, 2019 Nov 18.
Artigo em Inglês | MEDLINE | ID: mdl-31735908

RESUMO

BACKGROUND Current guidelines are inadequate for use in predicting ITP recurrence. Therefore, our primary goal in this study was to investigate the association of platelet-to-lymphocyte ratio (PLR) at diagnosis with ITP recurrence in Chinese patients. MATERIAL AND METHODS We performed a historical cohort study and non-selectively enrolled 233 patients with newly-identified ITP from March 2013 to June 2017. The independent variable was PLR recorded at diagnosis and the dependent variable was recurrence-free survival (RFS) at 6 months. Data on the following variables were also collected for establishing a multivariate Cox regression model: demographic details, general details, and variables found to be closely related to PLR in previous studies, as well as risk factors for ITP recurrence. RESULTS During follow-up, 85 patients had an event within 6 months. At the range of 0.86-9.7 of PLR, a 1-unit increase in PLR was associated with a 13% decrease in ITP recurrence (hazard ratio: 0.87; 95% confidence interval: 0.78-0.97), whereas no association was detected at the range of 9.7-33.75 of PLR (hazard ratio: 0.99; 95% confidence interval: 0.95-1.04). An interaction test indicated that patients with HP infection (0.91 (0.86-1.97)) or diabetes history (0.86 (0.78-0.96)) showed a stronger association compared with patients without HP infection (1.01 (0.95-1.04) and those without diabetes (1.01 (0.97-1.04)). CONCLUSIONS Our findings suggest that PLR is a useful parameter to consider when hematologists attempt to assess the risk of recurrence in ITP patients receiving first-line therapy, and the nonlinearity of PLR and ITP recurrence risk must be fully considered when constructing predictive models.


Assuntos
Plaquetas/citologia , Linfócitos/citologia , Púrpura Trombocitopênica Idiopática/metabolismo , Adolescente , Adulto , Idoso , Idoso de 80 Anos ou mais , China , Estudos de Coortes , Intervalo Livre de Doença , Feminino , Humanos , Contagem de Linfócitos , Masculino , Pessoa de Meia-Idade , Contagem de Plaquetas , Prognóstico , Modelos de Riscos Proporcionais , Púrpura Trombocitopênica Idiopática/sangue , Recidiva , Estudos Retrospectivos , Trombocitopenia/sangue , Trombocitopenia/metabolismo
8.
Medicine (Baltimore) ; 98(43): e17608, 2019 Oct.
Artigo em Inglês | MEDLINE | ID: mdl-31651870

RESUMO

This study aims to investigate the changes of cytokines and the effect of programmed death ligand 1 (PD-L1) signaling pathway on T cell function in patients with immune thrombocytopenic purpura (ITP).Totally, 40 untreated ITP patients were recruited and 30 healthy people were recruited as the healthy control. Then whole blood of ITP patients and healthy control was collected, respectively. The sPD-L1/anti-PD-1 was used to activate or block the programmed death (PD-1)/PD-L1 signaling pathway. The expression of PD-1 and PD-L1 on peripheral blood mononuclear cells (PBMCs) were detected by flow cytometry. PBMCs were treated with cluster of differentiation (CD3), cluster of differentiation 28 (CD28), and phytohaemagglutinin (PHA) for 48 hours. Serum levels of sPD-1, sPD-L1, and cytokines were measured by enzyme-linked immunosorbent assay (ELISA).Compared with the healthy control group, the percentages of PD-1+CD3+CD4+ T cells and PD-L1+HLA-DR+CD11c+ DC cells were increased in ITP patients. The levels of interferon-gamma (IFN-γ), interleukin-17 (IL-17), and sPD-1 in the serum of ITP patients were increased, while IL-4 and transforming growth factor-ß (TGF-ß) were decreased. Additionally, the level of sPD-1 was negatively correlated with the platelet count. Consistently, after treatment with CD3, CD28, and PHA, IFN-γ and IL-17 levels in culture supernatant of PBMCs from ITP patients were significantly higher than those from healthy controls whereas IL-4 and TGF-ß levels were significantly lower. Furthermore, IFN-γ and IL-17 levels secreted by PBMCs from ITP patients decreased after sPD-L1 administration, however, IL-4 and TGF-ß levels were increased. The level of IFN-γ in ITP group remained higher after anti-PD-1 blockage, but the levels of IL-4, TGF-ß, and IL-17 were not significantly influenced.sPD-1 may cause the dysfunction of PD-1/PD-L1 signaling pathway, and its level is related to the severity of ITP patients. Activation of PD-1/PD-L1 with sPD-L1 may restore the imbalance of Th1/Th2 and Treg/Th17 cell subtypes in ITP patients but anti-PD-1 may exacerbate disease by enhancing IFN-γ production.


Assuntos
Antígeno B7-H1/imunologia , Púrpura Trombocitopênica Idiopática/imunologia , Linfócitos T Reguladores/imunologia , Equilíbrio Th1-Th2/fisiologia , Células Th17/imunologia , Adulto , Antígenos CD28/imunologia , Complexo CD3/imunologia , Estudos de Casos e Controles , Citocinas/imunologia , Feminino , Humanos , Interferon gama/metabolismo , Leucócitos Mononucleares , Masculino , Pessoa de Meia-Idade , Fito-Hemaglutininas/imunologia , Receptor de Morte Celular Programada 1/fisiologia , Púrpura Trombocitopênica Idiopática/sangue , Transdução de Sinais/imunologia , Linfócitos T/imunologia , Equilíbrio Th1-Th2/efeitos dos fármacos , Fator de Crescimento Transformador beta/metabolismo
9.
Med Sci Monit ; 25: 7321-7331, 2019 Sep 29.
Artigo em Inglês | MEDLINE | ID: mdl-31563921

RESUMO

BACKGROUND In China, evidence regarding to the association between platelet to lymphocyte ratio (PLR) and glucocorticoid (GC) resistance in participants with primary newly identified immune thrombocytopenia (ITP) is limited. We aimed to investigate whether PLR is independently linked with GC-resistant ITP. MATERIAL AND METHODS We non-selectively and consecutively collected 154 newly diagnosed ITPs. The start enrollment time and the end enrollment time were from March 2013 to June 2017. The independent and dependent variables were PLR measured at diagnosis and GC non-response. Other variables involved in the present work can be summarized as demographic data and factors that were correlated with PLR reported by published studies. Univariate and multivariate binary logistic regression model and sensitivity analysis were used to evaluate the associations between PLR and GC resistance. RESULTS After adjusting covariates, PLR level was negatively associated with GC non-response [odds ratio (OR)=0.89, 95% confidence intervals (CI): 0.80 to 0.98], and supported by propensity score matching model (OR=0.74, 95%CI: 0.57 to 0.96]. Nonlinearity of PLR and GC resistance was observed whose inflection point was 5.08 (by 2-piecewise model). The OR and 95%CI on both sides of inflection point were 3.14 (0.81 to 12.21) and 0.81 (0.69 to 0.95), respectively. Subgroup analysis showed no significant differences from subgroups. CONCLUSIONS Threshold effect on PLR and GC resistance is observed. When PLR is larger than 5.08, a unit increase of PLR is independently associated with 19% reduction of GC resistance.


Assuntos
Púrpura Trombocitopênica Idiopática/metabolismo , Púrpura Trombocitopênica Idiopática/fisiopatologia , Adulto , Idoso , Plaquetas , China , Estudos de Coortes , Feminino , Humanos , Contagem de Linfócitos , Linfócitos , Masculino , Erros Inatos do Metabolismo , Pessoa de Meia-Idade , Neutrófilos , Ativação Plaquetária/fisiologia , Contagem de Plaquetas , Púrpura Trombocitopênica Idiopática/sangue , Curva ROC , Receptores de Glucocorticoides/deficiência , Estudos Retrospectivos
10.
Br J Hosp Med (Lond) ; 80(9): 507-512, 2019 Sep 02.
Artigo em Inglês | MEDLINE | ID: mdl-31498668

RESUMO

Thrombocytopenia is defined as a platelet count under 150x109/litre. It may be found as a bystander to other pathology or directly related to an underlying haematological condition. Apart from laboratory artefact, it should be treated seriously as it often reflects serious underlying disease. This review uses short case histories to illustrate how to approach thrombocytopenia during the initial presentation of an adult patient to hospital. This article guides the general hospital physician through the narrow but potentially confusing differential diagnoses related to thrombocytopenia, with particular focus on immune thrombocytopenia, disseminated intravascular coagulation and thrombotic thrombocytopenic purpura. Thrombocytopenia in pregnancy deserves special consideration and will not be discussed in this article.


Assuntos
Coagulação Intravascular Disseminada/diagnóstico , Púrpura Trombocitopênica Idiopática/diagnóstico , Púrpura Trombocitopênica Trombótica/diagnóstico , Doença Aguda , Diagnóstico Diferencial , Coagulação Intravascular Disseminada/sangue , Coagulação Intravascular Disseminada/terapia , Produtos de Degradação da Fibrina e do Fibrinogênio/metabolismo , Fibrinogênio/metabolismo , Humanos , Coeficiente Internacional Normatizado , Tempo de Tromboplastina Parcial , Transfusão de Plaquetas/métodos , Púrpura Trombocitopênica Idiopática/sangue , Púrpura Trombocitopênica Idiopática/terapia , Púrpura Trombocitopênica Trombótica/sangue , Púrpura Trombocitopênica Trombótica/terapia , Trombocitopenia
11.
Ann Hematol ; 98(10): 2299-2302, 2019 Oct.
Artigo em Inglês | MEDLINE | ID: mdl-31444663

RESUMO

Iron deficiency anemia (IDA) is often associated with mild to moderate thrombocytosis, and iron deficiency-associated thrombocytopenia (IDAT) is much more uncommon and often misdiagnosed as immune thrombocytopenia (ITP). To better describe the features of IDAT, we conducted a retrospective multicenter case-control study. We identified 10 patients (9 women) with a definite diagnosis IDAT, with a median age of 43.5 [range, 16-72] years and a median platelet count of 30.5 × 109/L [range, 21-80], and 7 patients with a possible diagnosis of IDAT. Bleeding manifestations were absent in all patients but one. All the patients recovered (platelet count ≥ 150 × 109/L) upon iron therapy ± red blood cell transfusion after a median time of 6 [4-39] days. When compared with 30 randomly newly diagnosed ITP patients matched on age, the baseline platelet count was significantly lower in ITP (median = 7 × 109/L [4-59], p < 0.001) whereas MPV was higher (10.5 fL [9,4-13,8] vs 8.2 fL, for IDAT p < 0.001). The median platelet count on day 7 was 337 × 109/L [113-1000] for IDAT cases vs 72 × 109/L [13-212] for ITP controls (p < 0.001). IDAT is potentially an under-recognized cause of thrombocytopenia that may be easily managed with iron therapy.


Assuntos
Anemia Ferropriva , Trombocitopenia , Adolescente , Adulto , Fatores Etários , Idoso , Anemia Ferropriva/sangue , Anemia Ferropriva/complicações , Anemia Ferropriva/diagnóstico , Anemia Ferropriva/epidemiologia , Erros de Diagnóstico , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Contagem de Plaquetas , Púrpura Trombocitopênica Idiopática/sangue , Púrpura Trombocitopênica Idiopática/diagnóstico , Púrpura Trombocitopênica Idiopática/epidemiologia , Púrpura Trombocitopênica Idiopática/etiologia , Estudos Retrospectivos , Trombocitopenia/sangue , Trombocitopenia/diagnóstico , Trombocitopenia/epidemiologia , Trombocitopenia/etiologia
12.
Postgrad Med J ; 95(1128): 558-562, 2019 Oct.
Artigo em Inglês | MEDLINE | ID: mdl-31320499

RESUMO

Patients with immune thrombocytopaenia (ITP) have a wide spectrum of disease severity and bleeding risk even at similar platelet counts. Hence, additional clinical and laboratory factors may be considered in the evaluation of bleeding risk in ITP. Risk stratification based on predicted bleeding risk may help to identify high-risk patients and guide the initial management of ITP in adults requiring treatment. Recent evidence supports the use of high-dose dexamethasone therapy over prednisone in the initial management of ITP because of improved initial response rates, shorter median time to response and better safety profile. A risk-stratified approach to management of ITP is hoped to reduce bleeding complications in high-risk patients; however, the outcomes of such management approach need to be studied prospectively. Additionally, whether therapy intensification or combination of dual therapy such as intravenous immunoglobulin or rituximab in combination with dexamethasone can reduce bleeding complications in high-risk ITP should be studied in the future.


Assuntos
Glucocorticoides/administração & dosagem , Púrpura Trombocitopênica Idiopática/tratamento farmacológico , Medição de Risco , Adulto , Dexametasona/administração & dosagem , Relação Dose-Resposta a Droga , Resistência a Medicamentos , Hemorragia/etiologia , Hemorragia/prevenção & controle , Humanos , Volume Plaquetário Médio , Contagem de Plaquetas , Púrpura Trombocitopênica Idiopática/sangue
13.
Lupus ; 28(8): 986-994, 2019 Jul.
Artigo em Inglês | MEDLINE | ID: mdl-31246559

RESUMO

OBJECTIVE: We aimed to study the usefulness of serum soluble CD163 (sCD163) as a biomarker for macrophage activation syndrome (MAS) associated with systemic lupus erythematosus (SLE). METHODS: Serum sCD163 levels were retrospectively measured by enzyme-linked immunosorbent assay for SLE patients associated with MAS (SLE-MAS), lupus nephritis (LN), or autoimmune hemolytic anemia (AIHA) and/or immune thrombocytopenia (ITP) and healthy controls (HCs). Posttreatment samples were also evaluated in the available SLE-MAS patients. The associations between serum sCD163 levels and clinical information were statistically analyzed. RESULTS: The serum sCD163 levels in SLE-MAS, LN and SLE-AIHA/ITP groups were significantly higher than those in HCs (n = 17, 29, 13, and 68, respectively; p < 0.01 for all comparisons). In addition, the serum sCD163 levels in the SLE-MAS group were even higher than those in the LN and SLE-AIHA/ITP groups (p < 0.01 for both comparisons). Serum sCD163 levels were correlated with the SLE Disease Activity Index 2000 scores (r = 0.53), whereas they were not correlated with the serum ferritin levels. With the determined cut-off value, the sensitivity and specificity of serum sCD163 for the diagnosis of SLE-MAS were 59% and 86%, respectively. Retesting showed that the serum sCD163 levels decreased significantly following treatment in parallel with disease amelioration in the SLE-MAS group (p < 0.01). CONCLUSIONS: The present study suggests the usefulness of serum sCD163 as a diagnostic and disease-activity biomarker for SLE-associated MAS. Serum sCD163 might also have a different role as a biomarker for SLE-associated MAS than serum ferritin does.


Assuntos
Antígenos CD/sangue , Antígenos de Diferenciação Mielomonocítica/sangue , Lúpus Eritematoso Sistêmico/complicações , Síndrome de Ativação Macrofágica/sangue , Receptores de Superfície Celular/sangue , Adulto , Anemia Hemolítica Autoimune/sangue , Biomarcadores/sangue , Estudos de Casos e Controles , Ensaio de Imunoadsorção Enzimática , Feminino , Humanos , Lúpus Eritematoso Sistêmico/sangue , Nefrite Lúpica/sangue , Síndrome de Ativação Macrofágica/diagnóstico , Macrófagos/metabolismo , Masculino , Pessoa de Meia-Idade , Púrpura Trombocitopênica Idiopática/sangue , Curva ROC , Estudos Retrospectivos
14.
Ann Ital Chir ; 90: 417-420, 2019.
Artigo em Inglês | MEDLINE | ID: mdl-31203266

RESUMO

PURPOSE: Minimal invasive procedures has become increasingly popular during the last decades. The aim of this retrospective study was to evaluate the safety and feasibility of laparoscopic splenectomy in patients with immune thrombocytopenic purpura who has very low platelet counts. METHODS: Between March 28, 2005 and June 08, 2013, a total of 132 patients with the diagnosis of immune thrombocytopenic purpura were included to study. The patients who underwent laparoscopic splenectomy were alienated into two groups according to their platelet counts lower than 10000 (group 1) and higher than 10000 (group 2) RESULTS: There were 16 patients in group 1 with very low platelet counts, and 116 in group 2. One patient in group 1 had converted to laparotomy due to peroperative bleeding, and there were 5 conversion to open in group 2. There were also 2 patients in group 2 who underwent laparatomy on post operative day 1 due to delayed intra-abdominal bleeding. Moreover, one patient in each group had pancreatic fistula. CONCLUSIONS: Laparoscopic splenectomy is a safe technique in patients with ITP even the patients have very low platelet counts. KEY WORDS: ITP, Laparoscopy, Low platelet count, Splenectomy.


Assuntos
Laparoscopia/métodos , Contagem de Plaquetas , Púrpura Trombocitopênica Idiopática/cirurgia , Esplenectomia/métodos , Adulto , Idoso , Perda Sanguínea Cirúrgica , Conversão para Cirurgia Aberta/estatística & dados numéricos , Estudos de Viabilidade , Feminino , Humanos , Laparoscopia/efeitos adversos , Masculino , Pessoa de Meia-Idade , Fístula Pancreática/etiologia , Complicações Pós-Operatórias/etiologia , Hemorragia Pós-Operatória/etiologia , Púrpura Trombocitopênica Idiopática/sangue , Estudos Retrospectivos , Esplenectomia/efeitos adversos , Resultado do Tratamento , Adulto Jovem
15.
Pediatr Blood Cancer ; 66(9): e27874, 2019 09.
Artigo em Inglês | MEDLINE | ID: mdl-31207059

RESUMO

Growth factor-independent 1B (GFI1B) variants are a rare cause of thrombocytopenia. We report on a male child who was initially diagnosed with immune thrombocytopenia. However, subtle clinical signs led to suspicion of a genetic cause of thrombocytopenia. Gene panel sequencing revealed a rare variant in GFI1B (C168F), which has recently been reported in several families with thrombocytopenia. We demonstrate that this variant significantly alters platelet parameters in population studies. This case highlights how diagnoses of exclusion, such as immune thrombocytopenia, can be confounded by genetic variation. Our understanding of blood disorders will undoubtedly evolve from an increased knowledge of human genetic variation.


Assuntos
Plaquetas/metabolismo , Doenças Genéticas Inatas , Mutação de Sentido Incorreto , Proteínas Proto-Oncogênicas/genética , Púrpura Trombocitopênica Idiopática , Proteínas Repressoras/genética , Pré-Escolar , Doenças Genéticas Inatas/sangue , Doenças Genéticas Inatas/diagnóstico , Doenças Genéticas Inatas/genética , Humanos , Masculino , Púrpura Trombocitopênica Idiopática/sangue , Púrpura Trombocitopênica Idiopática/diagnóstico , Púrpura Trombocitopênica Idiopática/genética
16.
Transfus Apher Sci ; 58(4): 491-494, 2019 Aug.
Artigo em Inglês | MEDLINE | ID: mdl-31105060

RESUMO

OBJECTIVES: Assess the appropriateness of the use of intravenous immunoglobulin (IVIG) for immune thrombocytopenia (ITP). BACKGROUND: IVIG is suggested for ITP when a rapid increase in platelet count is desired or as first line therapy if corticosteroids are contraindicated. A recent Canadian audit of IVIG requests found a lack of compliance with provincial requirements and inadequate documentation of efficacy which led the authors to conclude that the use of IVIG was broadly inappropriate for all treated diseases. METHODS: Retrospective review of patients with ITP who received IVIG at our institution over a one-year period. RESULTS: 40 patients received IVIG for ITP over the study period for a total of 76 infusions. The most common indications for IVIG within currently accepted guidelines were: active bleeding (13, 17%), pre-operative or antepartum care (22, 29%), contraindication to corticosteroids (8, 11%), and requirement for antithrombotic or myelosuppressive therapy (5, 7%). Indications that fell outside of guidelines included use of IVIG as a diagnostic challenge where the etiology of thrombocytopenia was unclear. IVIG was generally well tolerated. CONCLUSION: At our institution, use of IVIG for ITP was generally appropriate and carefully considered. Detailed utilization/knowledge data inquiries are required to develop tools and policies to enhance appropriate IVIG use in multiple settings.


Assuntos
Imunoglobulinas Intravenosas/administração & dosagem , Auditoria Médica , Púrpura Trombocitopênica Idiopática/tratamento farmacológico , Administração Intravenosa , Adulto , Canadá/epidemiologia , Feminino , Humanos , Masculino , Púrpura Trombocitopênica Idiopática/sangue , Púrpura Trombocitopênica Idiopática/epidemiologia , Estudos Retrospectivos
18.
Haematologica ; 104(6): 1112-1123, 2019 06.
Artigo em Inglês | MEDLINE | ID: mdl-31073079

RESUMO

The two thrombopoietin receptor agonists (TPO-RA), eltrombopag and romiplostim, were licensed in the US for treatment of immune thrombocytopenia (ITP) in 2008 and, since then, their use has progressively increased around the world; they are currently used in more than 100 countries. The six largest randomized controlled trials conducted in ITP have used one of these two agents. All studies have demonstrated a platelet response rate between 50-90%, depending on the criteria used, with good safety and tolerability. TPO-RA were shown to be effective in reducing bleeding and the need for concomitant or rescue medication. Many other investigations of their mechanism of effect, prospective and retrospective trials, and studies focusing on toxicity have been performed widening our knowledge of these two agents. Initial concerns on issues such as myelofibrosis have not been confirmed. Only a small number of patients develop moderate-severe reticulin fibrosis and/or collagen fibrosis; however, these are usually reversed after discontinuation of TPO-RA. Studies indicate, however, that TPO-RA may increase the risk of venous thromboembolism. Both TPO-RA are currently approved in patients with chronic ITP aged >1-year who are refractory to at least one other treatment. Eltrombopag has acquired two additional indications: severe aplastic anemia refractory to first-line treatment and hepatitis C patients undergoing treatment with interferon-ribavirin. Despite these wide-ranging studies, important questions still need to be answered. This summary review on TPO-RA will summarize what is known regarding efficacy in ITP, evaluate safety concerns in more depth, and focus on the questions that remain.


Assuntos
Benzoatos/uso terapêutico , Hidrazinas/uso terapêutico , Pirazóis/uso terapêutico , Receptores Fc/uso terapêutico , Receptores de Trombopoetina/agonistas , Proteínas Recombinantes de Fusão/uso terapêutico , Trombopoetina/uso terapêutico , Animais , Benzoatos/química , Benzoatos/farmacologia , Biomarcadores , Coagulação Sanguínea/efeitos dos fármacos , Ensaios Clínicos como Assunto , Suscetibilidade a Doenças , Humanos , Hidrazinas/química , Hidrazinas/farmacologia , Púrpura Trombocitopênica Idiopática/sangue , Púrpura Trombocitopênica Idiopática/complicações , Púrpura Trombocitopênica Idiopática/tratamento farmacológico , Púrpura Trombocitopênica Idiopática/etiologia , Pirazóis/química , Pirazóis/farmacologia , Receptores Fc/química , Receptores de Trombopoetina/química , Receptores de Trombopoetina/metabolismo , Proteínas Recombinantes de Fusão/química , Proteínas Recombinantes de Fusão/farmacologia , Transdução de Sinais/efeitos dos fármacos , Trombopoetina/química , Trombopoetina/farmacologia , Resultado do Tratamento
19.
Exp Hematol ; 73: 18-24, 2019 05.
Artigo em Inglês | MEDLINE | ID: mdl-31014934

RESUMO

Indirubin, a traditional Chinese medicine, is currently used to treat certain autoimmune diseases such as primary immune thrombocytopenia (ITP) in clinics. However, the effects of indirubin on expression of related genes in peripheral blood mononuclear cells (PBMCs) from ITP patients have not been investigated. In the present study, PBMCs were isolated from 19 adult patients with well-characterized active ITP and 20 healthy controls (HCs) and then treated with increasing concentrations of indirubin. The mRNA expression levels of thrombopoietin receptor (MPL), GATA binding protein 3 (GATA3), DNA methyltransferase 3B (DNMT3B), interleukin-6 (IL6), tumor necrosis factor (TNF), and interferon gamma (IFN-γ) were determined by quantitative real-time polymerase chain reaction (PCR). We found that indirubin had no cytotoxic effect on PBMC viability. Significantly lower MPL (p < 0.05) and GATA3 (p < 0.05) expression together with markedly higher IL6 (p < 0.05), TNF (p < 0.0001), and IFN-γ (p < 0.001) mRNA levels were observed in ITP patients compared with HCs. Notably, indirubin significantly enhanced MPL expression and inhibited TNF expression in PBMCs from ITP patients (p < 0.05). In summary, indirubin may play a direct role in thrombopoiesis by activating cellular MPL and normalizing TNF expression to suppress inflammation in ITP. This study may thus improve our understanding of indirubin and provide important information for optimizing therapeutic strategies for ITP patients.


Assuntos
Regulação da Expressão Gênica/efeitos dos fármacos , Leucócitos Mononucleares/metabolismo , Púrpura Trombocitopênica Idiopática , Receptores de Trombopoetina/sangue , Fator de Necrose Tumoral alfa/sangue , Adulto , Idoso , DNA (Citosina-5-)-Metiltransferases/sangue , Feminino , Fator de Transcrição GATA3/sangue , Humanos , Indóis/administração & dosagem , Interferon gama/sangue , Interleucina-6/sangue , Leucócitos Mononucleares/patologia , Masculino , Pessoa de Meia-Idade , Púrpura Trombocitopênica Idiopática/sangue , Púrpura Trombocitopênica Idiopática/tratamento farmacológico , Púrpura Trombocitopênica Idiopática/patologia
20.
Medicine (Baltimore) ; 98(16): e15195, 2019 Apr.
Artigo em Inglês | MEDLINE | ID: mdl-31008943

RESUMO

This study evaluated the effectiveness of recombinant human interleukin-11 (rhIL-11) in the treatment of immune thrombocytopenia (ITP) and determined whether clinical and laboratory findings predicted the treatment response.This retrospective, single-center, case-control study included 103 adult patients with ITP treated between July 2010 and April 2014 at Jiangxi Province People's Hospital. About 49 patients in the pred+IL group received prednisone (conventional dose) combined with an rhIL-11 regimen, and 54 patients in the pred alone group received prednisone (conventional dose) alone. Demographic data, initial and follow-up platelet counts, proportions of patients achieving platelet counts ≥30 × 10/L (response) and ≥100 × 10/L (complete response) at different time points, and adverse reactions were compared between groups.Complete response rates were similar between groups overall but higher in the pred+IL group than in the pred alone group for newly diagnosed patients and those with severe ITP (P < .05). Proportions of patients achieving response or complete response at different time points were similar between groups overall but higher in the pred+IL group than in the pred alone group for newly diagnosed patients and those with severe ITP (P < .05). Posttreatment platelet count correlated negatively with platelet count at diagnosis and white blood cell (WBC) count at diagnosis in patients with newly diagnosed ITP (r = -0.337, P = .073 and r = -0.367, P = .050, respectively) or ITP with bleeding-related episodes (r = -0.357, P = .020 and r = -0.434, P = .004, respectively). No immediate or postinfusion severe adverse reactions were observed.rhIL-11 increased CR and improved hemostasis in patients with newly diagnosed or severe ITP. Platelet and WBC counts at diagnosis can predict the response to rhIL-11.


Assuntos
Antineoplásicos/uso terapêutico , Interleucina-11/uso terapêutico , Púrpura Trombocitopênica Idiopática/tratamento farmacológico , Adolescente , Adulto , Idoso , Feminino , Humanos , Contagem de Leucócitos , Masculino , Pessoa de Meia-Idade , Contagem de Plaquetas , Prednisona/uso terapêutico , Púrpura Trombocitopênica Idiopática/sangue , Proteínas Recombinantes/uso terapêutico , Estudos Retrospectivos , Adulto Jovem
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