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1.
Zhonghua Xue Ye Xue Za Zhi ; 45(3): 264-270, 2024 Mar 14.
Artigo em Chinês | MEDLINE | ID: mdl-38716598

RESUMO

Objective: To evaluate the efficacy and safety of roxadustat in patients with refractory non-severe aplastic anemia (NSAA) . Methods: The clinical data of patients with refractory NSAA who had been treated with roxadustat continuously for at least 3 months and followed up for more than 6 months at Peking Union Medical College Hospital from October 2020 to August 2022 were retrospectively collected. The demographic information, clinical data, treatment efficacy, adverse reactions, and outcomes were evaluated, and the factors influencing efficacy were analyzed. Results: A total of 41 patients were included. The male-to-female ratio was 16∶25, and the median age was 52 (18-84) years. The median duration of roxadustat treatment was 5 (3-20) months, and the median follow-up was 15 (6-26) months. Hematologic improvement-erythroid (HI-E) was 12.2%, 29.3%, 46.3%, 43.9%, and 30.3% at 1, 2, 3, 6, and 12 months, respectively. The rate of transfusion independence was 28.5%, 38.1%, and 33.3% at 3, 6, and 12 months, respectively. Hemoglobin returned to normal in some patients after treatment with roxadustat. The incidence of adverse events was 22%, all of which were grade Ⅰ-Ⅱ and recoverable. No factors that could affect HI-E were identified. By the end of follow-up, 45% of the patients relapsed, with a median time to relapse of 7 (3-12) months. No clonal evolution was observed, and one patient died. Conclusion: Roxadustat effectively improved anemia with good tolerance in patients with refractory NSAA.


Assuntos
Anemia Aplástica , Glicina , Isoquinolinas , Humanos , Masculino , Feminino , Anemia Aplástica/tratamento farmacológico , Pessoa de Meia-Idade , Adulto , Estudos Retrospectivos , Idoso , Adolescente , Isoquinolinas/uso terapêutico , Isoquinolinas/efeitos adversos , Glicina/análogos & derivados , Glicina/uso terapêutico , Glicina/efeitos adversos , Resultado do Tratamento , Idoso de 80 Anos ou mais , Adulto Jovem
2.
Hematology ; 29(1): 2298523, 2024 Dec.
Artigo em Inglês | MEDLINE | ID: mdl-38156735

RESUMO

BACKGROUND: Cyclosporine A (CsA) and regular doses of recombinant human thrombopoietin (rhTPO) can accelerate platelet recovery in patients with non-severe aplastic anemia (NSAA). However, it is unclear whether CsA plus rhTPO at a higher dose can further increase the efficacy. METHODS: Data from patients with newly diagnosed NSAA, who had been treated with CsA in combination with different doses of rhTPO between February 2021 and August 2021 at Peking Union Medical College Hospital, were reviewed. All the enrolled patients had been treated with CsA at 3-5 mg/(kg/d), and patients were further classified into high-dose (with rhTPO 30000U qd × 14 days for 2 months) group or regular-dose (with rhTPO 15000U qd × 7days for 3 months) group. The treatment response and therapy-related adverse events were compared. RESULTS: 36 patients including 16 (44.4%) in the high-dose and 20 (55.6%) in the regular-dose group were enrolled. The baseline characteristics were compatible between the two groups. The platelet counts were significantly higher at 1/3/6 months in the high-dose group (p = 0.028, 0.0063 and p = 0.040, respectively). The high-dose group had a significantly shorter time to platelet transfusion independence ([1 (0.5-6) months vs 2.5 (1-12) months, p = 0.040]). There was no significant difference in overall response and complete response rate between the two groups at 1/3/6/12 months (p > 0.05). Treatment-related morbidities were similar between the two groups (p > 0.05). CONCLUSIONS: Adding a higher dose of rhTPO can further accelerate platelet recovery and platelet transfusion independence in patients with newly diagnosed NSAA.


Assuntos
Anemia Aplástica , Humanos , Anemia Aplástica/tratamento farmacológico , Ciclosporina/uso terapêutico , Trombopoetina/efeitos adversos , Estudos Retrospectivos , Contagem de Plaquetas , Proteínas Recombinantes/efeitos adversos
3.
Ann Med ; 55(1): 2224044, 2023 12.
Artigo em Inglês | MEDLINE | ID: mdl-37318085

RESUMO

INTRODUCTION: The therapeutic options for thrombocytopenia in non-severe aplastic anaemia (NSAA) are limited. Avatrombopag (AVA) is prescribed for thrombocytopenic diseases but not for NSAA. METHODS: Herein, we conducted a phase 2, non-randomized, single-arm trial to explore the efficacy and safety of AVA in refractory/relapsed/intolerant NSAA. AVA dose was initiated at 20 mg/d and titrated to a maximum of 60 mg/d. The primary endpoint was the haematological response at 3 months. RESULTS: Twenty-five patients were analyzed. The overall response rate (ORR) at 3 months was 56% (14/25), with 12% (3/25) achieving a complete response (CR). At a median follow-up of 7 (3-10) months, the OR and CR rates were 52% and 20%, respectively. Responders had a shorter duration of diagnosis of AVA administration than non-responders (10 (6-80) vs 37 (6-480) months, p = 0.027) and belonged to the relapsed/intolerant NSAA type (71% vs 27%, p = 0.047); 44% (8/18) patients previously treated with eltrombopag before enrollment responded at 3 months, with an average prior eltrombopag dose of median 72.5 (50-100) mg/d and an average AVA dose for a response of median 43.5 (20-60) mg/d. 3-month ORR had no significant correlation with eltrombopag exposure (p = 0.09), prior eltrombopag length (R2=0.11), or cumulative eltrombopag dose (R2=0.30). Only one patient relapsed after stopping AVA for 1 month. No serious AVA-related side effects or clone evolution were detected. CONCLUSION: AVA is effective and well-tolerated in NSAA patients who are refractory, relapsed, or intolerant to CsA/tacrolimus ± eltrombopag. Earlier treatment and relapsed/intolerant AA may show a better short-term response rate. More studies are needed to define the optimal dose and the long-term efficacy (NCT04728789).


Assuntos
Anemia Aplástica , Humanos , Anemia Aplástica/tratamento farmacológico , Receptores de Trombopoetina , Pirazóis/efeitos adversos
4.
Zhongguo Shi Yan Xue Ye Xue Za Zhi ; 31(3): 823-829, 2023 Jun.
Artigo em Chinês | MEDLINE | ID: mdl-37356946

RESUMO

OBJECTIVE: To compare the efficacy of eltrombopag combined with cyclosporine A (CsA) and CsA alone in patients with transfusion-dependent non-severe aplastic anemia (TD-NSAA). METHODS: The clinical data of 76 patients with treatment-naive TD-NSAA in Ningde Municipal Hospital of Ningde Normal University and Affiliated Hospital of Nantong University from December 2017 to June 2021 were retrospectively analyzed. Among them, 45 cases were treated with eltrombopag combined with CsA, and 31 patients with compatible baseline characters were treated with CsA alone. The efficacy of patients between the two groups was compared, and the factors affecting the curative effects were also analyzed. RESULTS: There were significant differences in hematological response (HR) and complete response(CR) rates between the two groups at 3, 6, 12 months, and follow-up endpoint of treatment (P<0.05). With the prolongation of eltrombopag treatment time, the curative effect increased gradually, and the patients achieved more CR and HR rates by the end of the follow-up period. Simultaneously, with the increase in the maximum stable dose of eltrombopag, the HR rate increased gradually. The megakaryocyte count in eltrombopag group was higher than that in control at 6 and 12 months (P<0.05). Compared with the control group, the median time of platelet transfusion independence in eltrombopag group was more shorter (P=0.018), and the median platelets transfusion volume was lower (P=0.009). At 3, 6, 12 months after eltrombopag, the change of platelet in eltrombopag group was higher than that in the control group (P<0.05). Analysis of related factors affecting the efficacy showed that sex, age, iron overload, platelet count before treatment had no effect on the efficacy, and the median maximum stable dosage and the administration period for eltrombopag were related to the curative effect. The patients of eltrombopag group experienced adverse events of varying degrees, but the reactions were mild and mostly tolerated. CONCLUSION: Eltrombopag can effectively improve the hematopoietic response and promote platelet recovery for TD-NSAA patients with relatively more residual hematopoietic cells, and it is safe and well tolerated.


Assuntos
Anemia Aplástica , Humanos , Anemia Aplástica/terapia , Estudos Retrospectivos , Resultado do Tratamento , Ciclosporina/uso terapêutico , Terapia de Imunossupressão , Imunossupressores/uso terapêutico
5.
Artigo em Chinês | WPRIM (Pacífico Ocidental) | ID: wpr-982136

RESUMO

OBJECTIVE@#To compare the efficacy of eltrombopag combined with cyclosporine A (CsA) and CsA alone in patients with transfusion-dependent non-severe aplastic anemia (TD-NSAA).@*METHODS@#The clinical data of 76 patients with treatment-naive TD-NSAA in Ningde Municipal Hospital of Ningde Normal University and Affiliated Hospital of Nantong University from December 2017 to June 2021 were retrospectively analyzed. Among them, 45 cases were treated with eltrombopag combined with CsA, and 31 patients with compatible baseline characters were treated with CsA alone. The efficacy of patients between the two groups was compared, and the factors affecting the curative effects were also analyzed.@*RESULTS@#There were significant differences in hematological response (HR) and complete response(CR) rates between the two groups at 3, 6, 12 months, and follow-up endpoint of treatment (P<0.05). With the prolongation of eltrombopag treatment time, the curative effect increased gradually, and the patients achieved more CR and HR rates by the end of the follow-up period. Simultaneously, with the increase in the maximum stable dose of eltrombopag, the HR rate increased gradually. The megakaryocyte count in eltrombopag group was higher than that in control at 6 and 12 months (P<0.05). Compared with the control group, the median time of platelet transfusion independence in eltrombopag group was more shorter (P=0.018), and the median platelets transfusion volume was lower (P=0.009). At 3, 6, 12 months after eltrombopag, the change of platelet in eltrombopag group was higher than that in the control group (P<0.05). Analysis of related factors affecting the efficacy showed that sex, age, iron overload, platelet count before treatment had no effect on the efficacy, and the median maximum stable dosage and the administration period for eltrombopag were related to the curative effect. The patients of eltrombopag group experienced adverse events of varying degrees, but the reactions were mild and mostly tolerated.@*CONCLUSION@#Eltrombopag can effectively improve the hematopoietic response and promote platelet recovery for TD-NSAA patients with relatively more residual hematopoietic cells, and it is safe and well tolerated.


Assuntos
Humanos , Anemia Aplástica/terapia , Estudos Retrospectivos , Resultado do Tratamento , Ciclosporina/uso terapêutico , Terapia de Imunossupressão , Imunossupressores/uso terapêutico
6.
Hematology ; 27(1): 733-741, 2022 Dec.
Artigo em Inglês | MEDLINE | ID: mdl-35688457

RESUMO

BACKGROUND: Whether combined CsA with androgen therapy was superior to androgen therapy alone in NSAA remains controversial. This study aimed to assess the efficacy and safety of combined therapy versus androgen therapy for NSAA patients using a meta-analytic approach. METHODS: An electronic database of PubMed, EmBase, Cochrane library, CNKI, VIP, and Wanfang was systematically searched for randomized controlled trials (RCTs) from their inception to February 2020. The primary endpoint was effective rate, while the secondary endpoints included white blood cell (WBC), hemoglobin, platelet, and potential adverse events. The pooled results from included trials were calculated with the random-effects model. RESULTS: Forty-three RCTs recruited 2610 NSAA patients for the final quantitative meta-analysis. We noted that combined therapy was associated with an increased incidence of effective rate than androgen therapy alone (relative risk [RR]: 1.35; 95% confidence interval [CI]: 1.29-1.41; P < 0.001). Moreover, patients treated with combined therapy were associated with higher WBC (weighted mean difference [WMD]: 1.22; 95%CI: 0.94-1.49; P < 0.001), hemoglobin (WMD: 12.93; 95%CI: 8.86-17.01; P < 0.001), and platelet (WMD: 8.65; 95%CI: 7.05-10.24; P < 0.001). Finally, the pooled incidence of hirsutism, handshake, gingiva hyperplasia, liver function damage, and renal function damage were 0.35 (95%CI: 0.22-0.48), 0.24 (95%CI: 0.15-0.32), 0.22 (95%CI: 0.10-0.35), 0.19 (95%CI: 0.14-0.25), and 0.06 (95%CI: 0.01-0.11), respectively. CONCLUSIONS: This study found that combined CsA with androgen therapy was superior to androgen therapy alone for Chinese patients with NSAA, and the most common adverse of combined therapy included hirsutism, handshake, gingiva hyperplasia, liver function damage, and renal function damage.


Assuntos
Anemia Aplástica , Ciclosporina , Adulto , Androgênios/efeitos adversos , Anemia Aplástica/tratamento farmacológico , Ciclosporina/efeitos adversos , Hirsutismo , Humanos , Hiperplasia , Ensaios Clínicos Controlados Aleatórios como Assunto
7.
Pediatr Transplant ; 26(7): e14340, 2022 11.
Artigo em Inglês | MEDLINE | ID: mdl-35735259

RESUMO

BACKGROUND: Non-severe aplastic anemia is more likely to develop into severe aplastic anemia, and there is no widely accepted treatment plan at present. Hematopoietic stem cell transplantation might be a new therapeutic strategy. METHODS: Retrospectively analyzed 32 patients with non-severe aplastic anemia who underwent hematopoietic stem cell transplantation from September 2007 to September 2020, and the 5-year estimated overall survival rate and the incidence of graft-versus-host disease were analyzed to evaluate the efficacy and safety of hematopoietic stem cell transplantation in the treatment of pediatric non-severe aplastic anemia. RESULTS: Thirty-two patients who underwent transplantation, 29 patients (90.6%) survived, 3 patients (9.4%) died. The incidence of acute graft-versus-host disease was 51.6% (16/31), including 15 cases (48.4%) of grade I-II and 1 case (3.2%) of grade III-IV. The incidence of chronic graft-versus-host disease was 38.7% (12/31). The 5-year overall survival rate was 91.8%. CONCLUSIONS: Hematopoietic stem cell transplantation is a practicable, safe, and effective treatment option for non-severe aplastic anemia pediatric patients who are suitable for transplant.


Assuntos
Anemia Aplástica , Doença Enxerto-Hospedeiro , Transplante de Células-Tronco Hematopoéticas , Anemia Aplástica/terapia , Criança , Doença Enxerto-Hospedeiro/etiologia , Humanos , Estudos Retrospectivos , Condicionamento Pré-Transplante
8.
Front Med (Lausanne) ; 9: 805197, 2022.
Artigo em Inglês | MEDLINE | ID: mdl-35342744

RESUMO

Objective: The management of children with non-severe aplastic anemia (NSAA) is undefined and the efficacies and benefits of immunosuppressive therapy remain inconsistent. The study aimed to investigate the efficacy of Cyclosporine (CsA) monotherapy for pediatric NSAA. Methods: Clinical data of children with NSAA who had been treated with CsA monotherapy at the outpatient department of Beijing Children's Hospital, Capital Medical University, National Children's Medical Center from January 2017 to March 2021 was collected retrospectively. Patients who had been treated <1 years until the end of follow-up were excluded. Transfusion-independent NSAA was further divided into moderate NSAA and mild NSAA according to the degree of cytopenia. Progression was defined as the development of transfusion-dependent AA or SAA and relapse was considered when treatment failed after initial response. Results: A total of 95 pediatric patients with NSAA were enrolled in this study with 49 (51.6%) patients confirmed as mild NSAA, 38 (40%) as moderate NSAA and 8 (8.4%) as transfusion-dependent NSAA. The median treatment time of CsA was 22 (12-44) months. The overall response rate (ORR) was 57.9%, with 30.5% CR and 27.4% PR. Unexpectedly, patients with mild NSAA acquired lowest ORR (46.9%), then patients with moderate NSAA (63.2%), while 8 patients who were transfusion-dependent all had an active response to CsA. The granulocyte and megakaryocyte response was 46.9 and 55.8% respectively, while the erythrocyte response rate was as low as 22.5%. Univariate analyses revealed that patients with lower platelet count and higher interleukin 10 level predict an active response to CsA while higher level of fetal hemoglobin (HbF) tended to be a negative factor. Data of Treg cells before and after 1 year's treatment was available in a total number of 40 patients. Paired comparison found that the percentage of Treg cells in CD4+ T cells was decreased after 1 year's treatment of CsA (6.78 ± 2.72 vs. 5.23 ± 2.06, P = 0.001),both in responders and non-responders. The degree of decline in Treg cells between two distinctive response groups had no significant difference (P>0.05). With a median follow-up time of 22 months, 10.9% of responders relapsed and maintained NSAA while 27.5% of non-responders progressed to SAA or became transfusion-dependent. The overall progression rate was 11.6%. Conclusion: CsA monotherapy had heterogeneous effects in the treatment of children NSAA Treatment approaches should be hierarchical and individual in clinical. Patients with lower platelet count and higher interleukin 10 level predicted an active response to CsA. While higher level of fetal hemoglobin (HbF) tended to be a negative factor. The percentage of Treg cells in CD4+ T cells was decreased broadly after treatment.

9.
Artigo em Chinês | WPRIM (Pacífico Ocidental) | ID: wpr-933462

RESUMO

The main purpose of our study was to evaluate the efficacy and safety of eltrombopag plus cyclosporine A (CsA) in transfusion-dependent non-severe aplastic anemia(TD-NSAA). The clinical characteristics of 13 TD-NSAA patients who received initial treatment of eltrombopag plus CsA from 2019 to 2021 were retrospectively analyzed. The 3-month overall hematological response (OR) rate was 12/13. Until the end of follow-up, 12 patients responded, among whom 2 patients reached complete response (CR) and 9 patients reached partial response (PR) and 1 with HR. Paroxysmal nocturnal hemoglobinuria (PNH) developed in one patient at 6 months after treatment. Five of thirteen patients reported mild adverse reactions, which were all manageable. Compared with historical data, the combination of eltrombopag with CsA is an effective regimen in patients with TD-NSAA.

10.
Front Pharmacol ; 11: 582625, 2020.
Artigo em Inglês | MEDLINE | ID: mdl-33364958

RESUMO

Background and purpose: Eltrombopag (ELT) can be effective in the treatment of relapse/refractory aplastic anemia (AA) patients. Responses and adverse drug reactions (ADRs) differed greatly among individuals treated at the same dosage of ELT. Methods: Patients diagnosed with nonsevere aplastic anemia (NSAA) between January 2018 and January 2019 in Peking Union Medical Colleague Hospital who were refractory to immunosuppressive therapy were treated with ELT and followed up for at least 6 months. Plasma concentrations of ELT were detected by high-performance liquid chromatography-mass spectrometry after at least two months of ELT treatment and treatment at the same dosage for at least 2 weeks. The dose-concentration, concentration-response and concentration-ADR relationships were evaluated. Results: Among the 72 patients treated with ELT during the study period, 44 patients with complete data were enrolled. Six (13.6%) were males, and 38 were females (86.4%), with a median age of 54 years [interquartile range (IQR): 38.5-63]. At the time the ELT plasma concentration was detected, the median dosage of ELT was 75 (IQR 50-100) mg/d, the median time of total ELT exposure was 3 (IQR 2.0-6.0) months, and 37 (70.5%) patients had responded to ELT. The median concentration of ELT was 10.4 µg/ml (IQR 3.7-24.4 µg/ml). The concentration of ELT was positively correlated with the daily dose of ELT (r = 0.68, p < 0.001). Multivariate logistic regression analysis showed that the risk of inefficacy of ELT at a concentration between 11.2 and 15.2 µg/ml was 0.028-fold (95% CI: 0.001-0.864; p = 0.041) of that at a concentration between 3.2 and 7.2 µg/ml. The cutoff value for the concentration of ELT showing efficacy was 12.50 µg/ml according to the receiver operation characteristic curve. A higher risk of ADR was related to a longer total exposure to ELT (p = 0.012). Although the correlation was not significant, the odds ratio increased with the ELT concentration, suggesting that it was possible that an elevated risk of ADR was correlated with the ELT blood concentration. Conclusion: ELT is effective for the treatment of NSAA and has acceptable side effects. The plasma concentration of ELT was correlated with the dose and the effects of ELT.

11.
Drug Des Devel Ther ; 14: 5711-5719, 2020.
Artigo em Inglês | MEDLINE | ID: mdl-33408460

RESUMO

BACKGROUND: For symptomatic non-severe aplastic anemia (NSAA) patients who cannot afford anti-thymocyte globulin (ATG) or allogeneic hematopoietic stem cell transplantation (HSCT), tacrolimus (FK) may be an option if these patients do not respond or become tolerant to cyclosporine A (CsA). METHODS: We enrolled 101 NSAA patients who were refractory or intolerant to CsA with no chance of HSCT or ATG treatment and treated these patients with tacrolimus for at least 6 months, with follow-up for at least one year. RESULTS: The overall response rate (ORR) was 38.6% (complete response: 9.9%; partial response: 28.7%), and the median time to optimal response was 6 (3~10) months. Thirty-two (31.7%) cases had elevated creatinine levels. Eight (7.9%) cases had elevations in AST/ALT. A total of 25.6% (10/39) of patients relapsed at the end of follow-up. Age (P=0.0005), FK concentration (4.0~12 ng/mL, P=0.0005) and intolerance to CsA (P=0.012) were the independent risk factors for ORR. Treg cell levels pre-FK treatment were much lower than those of healthy controls (3.7±0.6% vs 6.8±0.7%, P=0.0004) but increased significantly after FK treatment (3.7±0.6% vs 7.1±0.8%, P=0.0039). CONCLUSION: Our data suggest that tacrolimus is a salvage treatment for patients with NSAA that is refractory or intolerant to CsA.


Assuntos
Anemia Aplástica/tratamento farmacológico , Imunossupressores/uso terapêutico , Tacrolimo/uso terapêutico , Adolescente , Adulto , Idoso , Idoso de 80 Anos ou mais , Ciclosporina/uso terapêutico , Tolerância a Medicamentos , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Estudos Retrospectivos , Adulto Jovem
12.
Hematology ; 24(1): 405-412, 2019 Dec.
Artigo em Inglês | MEDLINE | ID: mdl-30907293

RESUMO

OBJECTIVES: The mechanism of non-severe aplastic anemia (NSAA) is not clear. It may be different from severe aplastic anemia (SAA). CD56bright NK cells (regulatory NK cells) is a subgroup of NK cells that produce immunoregulatory cytokines and express high-affinity IL-2 receptor. To investigate CD56bright NK cells quantities and function in patients with NSAA and to explore how CD56bright NK cells participate in the progress of this disease. METHODS: In this study, we analyzed the quantitative and functional changes of CD56bright NK cells in peripheral blood of patients with NSAA by using Flow Cytometry (FCM) before and after immunosuppressive therapy (IST). The expressions of activating receptor (NKG2D, NKp46, NKp44), inhibitory receptor (NKG2A, CD158a, CD158b) and perforin and granzyme B were detected by FCM. IL-2 and IL-18 levels in serum were detected by ELISA. The correlation between these parameters and clinical indicators of patients were evaluated. RESULTS: We found that the percentage of CD56bright NK cells in newly diagnosed NSAA patients was higher than that in normal controls (p = .011, p < .05). The median expression of NKG2D in patients with NSAA was higher compared to that in normal controls (p = .021, p < .05), and the expression of CD158a was lower (p = .047, p < .05). The concentrations of IL-2 and IL-18 in the serum of patients with NSAA were higher than those in normal group. CONCLUSION: These findings suggest that increased and activated CD56bright NK cells might play a protective role in the pathogenesis of NSAA.


Assuntos
Anemia Aplástica/sangue , Anemia Aplástica/diagnóstico , Antígeno CD56/sangue , Células Matadoras Naturais/metabolismo , Adolescente , Adulto , Idoso , Anemia Aplástica/patologia , Criança , Feminino , Citometria de Fluxo , Humanos , Células Matadoras Naturais/patologia , Masculino , Pessoa de Meia-Idade
13.
Mol Med Rep ; 19(2): 1194-1202, 2019 02.
Artigo em Inglês | MEDLINE | ID: mdl-30569170

RESUMO

A type of aplastic anemia (AA), non-severe aplastic anemia (NSAA) is defined as AA that does not meet the diagnostic criteria of severe aplastic anemia (SAA). Complement component 1q (C1q) has an important role in the pathogenesis of various autoimmune diseases; however, the role of C1q in the immune pathogenesis of NSAA is not clear. The current study aimed to determine whether C1q has an important role in the pathogenesis of NSAA. Isobaric tags for relative and absolute quantitation (iTRAQ) was used to compare the protein expression in bone marrow mononuclear cells from patients with NSAA and healthy volunteers. Pathway enrichment analysis was performed to determine the biological functions involved in NSAA. The differential expression of C1q was marked compared with other proteins. Subsequently, the concentration of C1q in serum samples was determined using ELISA and the correlation of C1q levels and NSAA severity was evaluated. The serum concentrations of C1q were significantly lower in untreated patients with newly diagnosed NSAA compared with NSAA cases in remission and normal controls. Furthermore, there was no significant difference in C1q concentration between newly diagnosed patients with NSAA and patients with autoimmune hemolytic anemia or immune thrombocytopenia. The serum concentration of C1q in newly diagnosed NSAA was significantly lower in patients with SAA (P<0.0001); whereas, there was no significant difference between the patients with SAA, patients with NSAA remission and normal controls (P>0.05). Additionally, the serum C1q concentration was significantly correlated with granulocyte counts, the level of hemoglobin, platelet counts, reticulocyte percentage and remission in patients with NSAA. The serum C1q concentration was also positively correlated with the myeloid/plasmacytoid dendritic cell ratio, and negatively correlated with the CD4(+)/CD8(+) ratio. These findings suggested that C1q may be a reliable serological marker for monitoring and evaluating disease severity in patients with NSAA. C1q may have an important role in the immune pathogenesis of NSAA.


Assuntos
Anemia Aplástica/sangue , Anemia Aplástica/patologia , Complemento C1q/metabolismo , Adolescente , Adulto , Idoso , Anemia Aplástica/metabolismo , Biomarcadores/sangue , Células da Medula Óssea/metabolismo , Criança , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Índice de Gravidade de Doença , Adulto Jovem
14.
Chongqing Medicine ; (36): 3170-3173, 2014.
Artigo em Chinês | WPRIM (Pacífico Ocidental) | ID: wpr-455927

RESUMO

Objective To evaluated the application value of mean corpuscular volume(MCV) and red blood cell volume distribu-tion width(RDW) in predicting early treatment responses of non-severe aplastic anemia(NSAA) .Methods 101 cases of patients who were newly diagnosed with non-severe aplastic anemia and were treated with Cyclosporine (CsA)combined androgen therapy . Treatment before baseline MCV ,RDW value were measured ,treatment for 3 months ,6 months test routine blood ,reticulocyte indi-cators and treatment results were statistically analyzed and receiver operating characteristic (ROC) curve was used to estimate the sensitivity and specificity of MCV ,RDW in predicting early treatment results .Results The level of MCV and RDW in non-severe aplastic anemia were significantly higher than normal reference .the responded patients had significantly higher pretreatment base-line MCV value than those non-responded .especially at 6 month ,baseline MCV were 105 .10 fl and 98 .30 fl respectively (P=0 .025) .the responded patients had significantly lower pretreatment baseline RDW value than those non-responded .especially at 3 month .baseline RDW were 14 .60% and 16 .60% respectively(P=0 .001) .The cutoff level of MCV (100 fL) and RDW (15 .55% ) for the predicting 3 and 6 month treatment responses were established based on the ROC curve ,with degree of accurancy of MCV was 62 .4% ,61 .4% and RDW 70 .3% ,63 .4% respectively .compared the treatment efficacy acuity MCV ≥100 fL/RDW <15 .55%group was significantly better than MCV < 100 fL/RDW≥15 .55% group .Multivariate analysis showed that pretreatment RDW and absolute reticulocyte value was the early prognostic factor of NSAA treatment effect .84 patients with ARC ≥ 20 × 109/L , through RDW cutoff Layered compare treatment response :RDW<15 .55% group was significantly better than the RDW≥15 .55%group(P=0 .000) .Conclusion MCV can not serve as of a significant predictor of early treatment response in non-severe aplastic a-nemia .RDW can serve as of the bone marrow failure severity indicators and a significant predictor of early treatment response in non-severe aplastic anemia .The joint reticulocyte absolute value parameters ,which can more accurately predict treatment efficacy .

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