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1.
Journal of Experimental Hematology ; (6): 823-829, 2023.
Article in Chinese | WPRIM | ID: wpr-982136

ABSTRACT

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.


Subject(s)
Humans , Anemia, Aplastic/therapy , Retrospective Studies , Treatment Outcome , Cyclosporine/therapeutic use , Immunosuppression Therapy , Immunosuppressive Agents/therapeutic use
2.
Journal of Experimental Hematology ; (6): 532-538, 2023.
Article in Chinese | WPRIM | ID: wpr-982091

ABSTRACT

OBJECTIVE@#To investigate the recovery characteristics of T cell subsets in patients with severe aplastic anemia (SAA) who received haploid hematopoietic stem cell transplantation(HSCT) and its relationship with acute graft-versus-host disease(aGVHD).@*METHODS@#The clinical data of 29 SAA patients who received haploid hematopoietic stem cell transplantation in the department of hematology, Shanxi Bethune Hospital from June 2018 to January 2022 were retrospectively analyzed. The absolute counts of CD3+T, CD4+T, CD8+T lymphocytes and the ratio of CD4+T/CD8+T lymphocytes in all patients before transplantation, 14, 21, 30, 60, 90 and 120 days after transplantation were analyzed. The proportion of T lymphocytes was compared in the non-aGVHD group, the grade Ⅰ-Ⅱ aGVHD group and the grade III-IV aGVHD group.@*RESULTS@#The counts of all T cells in 27 patients were far below the normal level at 14 and 21 days after transplantation, but there was obvious heterogeneity. There was a certain relationship between T cell immune reconstitution and conditioning regimen, age, and immunosuppressive treatment before transplantation. CD3+T cells showed a steady upward trend at 30, 60, 90, and 120 days after transplantation, and returned to the normal levels at 120 days after transplantation; faster recovery of CD4+T cells was closely related to aGVHD, which was at 30, 60, 90, 120 days after transplantation showed a slow upward trend, and which was still far below the normal level of 120 days after transplantation. CD8+T cell counts began to recover at 14 and 21 days after transplantation, and the recovery was earlier than the CD4+T cells, and its recovery speed was rapid 30 and 60 days after transptantation, which showed an upward trend and exceeded the normal levels 90 days after transplantation. Since CD8+ T cells reconstituted quickly, while the CD4+ T cells reconstitution was slowly, which made the long-term CD4+T/CD8+T cell ratio after transplantation was inverted . Compared with the non-aGVHD group, the absolute counts of CD3+T, CD4+T, and CD8+T cells in the aGVHD group were significantly higher than those in the non-aGVHD group at each time period after transplantation. In the aGVHD group, grade Ⅲ-Ⅳ aGVHD occurred more frequently in the early post-transplantation period (within 14-21 days), the grade Ⅰ-Ⅱ aGVHD group mostly occurred within 30-90 days after transplantation, and CD3+T, CD4+T, CD8+T cell counts in the grade Ⅲ-Ⅳ aGVHD group were significantly higher than those in the grade Ⅰ-Ⅱ aGVHD group; and the greater the proportion of CD4+T, the more severe the degree of aGVHD.@*CONCLUSION@#The speed of T cell immune reconstitution after SAA haploid transplantation is different, which is related to the conditioning regimen, age, and immunosuppressive therapy before transplantation. The rapid recovery of CD4+ T cells is closely related to the occurrence of aGVHD.


Subject(s)
Humans , Anemia, Aplastic/therapy , CD8-Positive T-Lymphocytes , Retrospective Studies , Haploidy , Hematopoietic Stem Cell Transplantation , Graft vs Host Disease
3.
Cienc. Salud (St. Domingo) ; 7(1): [37], 2023. tab
Article in Spanish | LILACS | ID: biblio-1444344

ABSTRACT

Introducción: la anemia aplásica (AA) es una enfermedad poco frecuente, caracterizada por presentar una insuficiencia en la médula ósea y una pancitopenia, sin rastro de procesos mieloproliferativos o fibróticos. Objetivo: conocer los diversos tratamientos farmacológicos usados en la terapia inmunosupresora (IST) en la AA; adicionalmente, se profundizará en la respuesta de los pacientes frente a la IST y los mecanismos de acción de los fármacos utilizados. Metodología: se realizó una búsqueda sistemática en las bases de datos de literatura médica como PUBMED, British Medical Journal, New England Journal, entre otros. Se incluyeron artículos tanto en inglés como en español. Conclusiones: el manejo de la anemia aplásica continúa representando un reto para la medicina moderna; no obstante, se ha desarrollado un gran número de nuevas opciones terapéuticas para tratar a los pacientes.


Introduction: Aplastic anemia (AA) is a rare disease characterized by presenting bone marrow failure and pancytopenia, with no trace of myeloproliferative or fibrotic processes. Objective: To present the different pharmacological treatments used in immunosuppressive therapy (IST) in AA, in addition, the response of patients to IST and the mechanisms of action of the drugs used will be studied in depth. Methodology: A systematic search was carried out in medical literature databases such as PUBMED, British Medical Journal, New England Journal, among others. Articles in both English and Spanish were included. Conclusions: The management of aplastic anemia continues to represent a challenge for modern medicine; however, a large number of new therapeutic options have been developed to treat patients.


Subject(s)
Humans , Bone Marrow , Anemia, Aplastic/drug therapy , Immunosuppressive Agents
4.
Article in English | AIM | ID: biblio-1512894

ABSTRACT

Dyslipidaemia has been implicated in the pathophysiology of sickle cell disease (SCD) complications; hence its role requires further elucidation. Objectives: To investigate the relationship between disease severity and plasma lipid levels of patients with sickle cell anaemia. Methods: A cross-sectional study design was used for the survey. A total of 50 patients with sickle cell anaemia and 50 controls without SCD were recruited for the study. The clinical data and plasma lipid levels of lipids and haemoglobin parameters were analysed. Results: The majority of the participants were aged 18-25 years. Total plasma cholesterol and HDL-C were significantly lower in individuals with SCA compared with the controls (3.3±1.2 vs 4.2±1.2; p<0.001) and (1.3±0.5 vs 1.5±0.4; p = 0.038) respectively. Most patients with SCA had moderate disease severity (24; 48%). There was no statistically significant difference in the plasma levels of total cholesterol and HDL-C across the disease severity groups of SCA (p = 0.694 and 0.262). There was also no significant correlation between total cholesterol, HDL-C, and markers ofhaemolysis, haemoglobin F, and haemoglobin S levels. Conclusion: SCA is characterised by lower mean plasma TC and HDL than controls. However, no relationship was found between TC, HDL levels and SCD disease severity, markers of haemolysis, HbF and HbS levels. Further studies are required to ascertain the implications of plasma lipid levels in SCD


Subject(s)
Humans , Cholesterol , Anemia, Sickle Cell , Anemia, Aplastic , Lipids
5.
Rev. peru. med. exp. salud publica ; 39(3): 372-375, jul.-sep. 2022. tab
Article in Spanish | LILACS | ID: biblio-1410013

ABSTRACT

RESUMEN Saprochaete capitata es una causa rara de infección fúngica invasiva en pacientes inmunocomprometidos con alta mortalidad y resistencia antifúngica. Presentamos el caso de un niño de cinco años con diagnóstico de aplasia medular, sometido a trasplante de progenitores hematopoyéticos (TPH), que cursó con neutropenia febril persistente, dolor abdominal intenso, aparición de lesiones maculopapulares en piel y deterioro de la función renal. Se identificó la presencia de S. capitata, en hemocultivos transcatéter venoso central. Esta infección fúngica invasiva resulta ser rara, pero emergente y potencialmente mortal, en pacientes con neutropenia febril persistente y uso prolongado de dispositivos invasivos intravasculares como catéter venoso central.


ABSTRACT Saprochaete capitata is a rare cause of invasive fungal infection in immunocompromised patients with high mortality and antifungal resistance. We present the case of a 5-year-old boy with bone marrow aplasia, who underwent hematopoietic stem cell transplantation (HSCT) and presented persistent febrile neutropenia, abdominal pain, appearance of maculopapular lesions on the skin, and impaired renal function. The presence of S. capitata was identified by blood culture from a central venous catheter. This invasive fungal infection is rare but emergent and life-threatening, especially in immunocompromised patients with persistent febrile neutropenia and prolonged use of invasive devices such as central venous catheters.


Subject(s)
Humans , Male , Child, Preschool , Immunocompromised Host , Invasive Fungal Infections/microbiology , Geotrichosis/microbiology , Geotrichum/isolation & purification , Anemia, Aplastic/complications , Fatal Outcome , Invasive Fungal Infections/drug therapy , Geotrichosis/drug therapy , Antifungal Agents/therapeutic use
6.
Brasília; CONITEC; maio 2022.
Non-conventional in Portuguese | BRISA, LILACS, ColecionaSUS | ID: biblio-1377732

ABSTRACT

INTRODUÇÃO: A anemia aplástica (AA) é uma doença rara e com risco de vida devido à falha herdada ou adquirida da medula óssea para produzir células sanguíneas, levando à pancitopenia progressiva. O tratamento para AA é determinado por uma série de fatores, incluindo gravidade da pancitopenia, idade do paciente, disponibilidade de doadores de célulastronco hematopoética, fonte de célula-tronco hematopoética, disponibilidade de imunossupressão e acesso a terapias ideais. Eltrombopague, um agonista de trombopoietina (TPO) vem emergindo como uma opção de tratamento para AA grave associado a terapia imunossupressora padrão. INDICAÇÃO: Pacientes adultos com anemia aplástica grave. PERGUNTA: Eltrombopague associado à terapia imunossupressora padrão (imunoglobulina antitimócito [ATG] e ciclosporina) é eficaz, seguro e custo-efetivo no tratamento de pacientes adultos com AA grave, quando comparado à terapia imunossupressora padrão (ATG e ciclosporina) isolada no SUS? EVIDÊNCIAS CLÍNICAS: A partir de uma busca bibliográfica conduzida nas bases PubMed, EMBASE e Cochrane Reviews, um ensaio clínico randomizado (ECR) foi selecionado, fornecendo evidências sobre a eficácia e segurança do eltrombopague adicionado ao tratamento imunossupressor em pacientes adultos com AA grave. Os pacientes apresentaram uma resposta completa em três meses de 10% no Grupo A (controle) e 22% no Grupo B (intervenção) (OR: 3,2; IC 95%, 1,3 a 7,8; P=0,01). Aos seis meses, a taxa de resposta geral foi de 41% no Grupo A e 68% no Grupo B. Os tempos médios para a primeira resposta foram de 8,8 meses (Grupo A) e 3,0 meses (Grupo B). A adição de eltrombopague à terapia imunossupressora padrão não resultou em melhora significativa da sobrevida global. A taxa de sobrevida global em dois anos foi de 85% (IC95%:78 a 92) para o grupo controle e de 90% (IC95%: 82 a 97) para o grupo intervenção. No entanto, o eltrombopague adicionado à terapia imunossupressora padrão aumentou a sobrevida livre de eventos de 34% para 46% em dois anos por meio da redução da refratariedade inicial à imunossupressão. A incidência de eventos adversos graves foi semelhante nos dois grupos. Para os desfechos resposta hematológica, sobrevida global, sobrevida livre de eventos, qualidade de vida e eventos adversos, considerou-se moderada qualidade de evidência pelo GRADE. AVALIAÇÃO ECONÔMICA: Na análise de custo-efetividade, empregando árvore de decisão e considerando-se o desfecho Taxa de Resposta Global, comparou-se o tratamento padrão (TP) versus a adição de eltrombopague ao TP, em um horizonte temporal de um ano. Ao demonstrar menor custo e maior efetividade, eltrombopague + TP se mostrou custo-efetiva no tratamento da AAG em pacientes não elegíveis ao TCTH, considerando-se a RCEI de - R$ 287.140,99. Na análise de sensibilidade, variando-se os custos de ATG e de eltrombopague, os resultados mostraram-se estáveis nos cenários avaliados, continuando a demonstrar que a adição de eltrombopague ao TP é custo-efetiva. ANÁLISE DE IMPACTO ORÇAMENTÁRIO: Avaliando-se três cenários diferentes em cinco anos, e considerando uma abordagem epidemiológica e demanda aferida (dispensações de ciclosporina para AA no SUS), a ampliação de uso do eltrombopague pode gerar economia de recursos de até R$ 241,2 milhões, ou seja, 12% menor do que o valor gasto no cenário de referência. Entretanto, nos cenários que se pressupõe menor uso do eltrombopague, a economia de recursos é menor. No cenário 2 o impacto orçamentário incremental é de - R$ 188,59 milhões e no cenário 3 é de - R$ 134 milhões. MONITORAMENTO DO HORIZONTE TECNOLÓGICO: Foi detectada uma tecnologia para compor o tratamento adicional a imunossupressor em pacientes adultos com AAG. Trata-se do hetrombopag, um agonista do receptor de trombopoetina, mesmo mecanismo de ação da tecnologia em avaliação neste relatório, encontrando-se em fase 3 de pesquisa clínica, e sem registro para qualquer indicação na Anvisa, FDA ou EMA. CONSIDERAÇÕES FINAIS: A AA é uma doença rara e frequentemente grave ou muito grave e que até o momento, não dispunha de um tratamento clínico com resultados satisfatórios, cenário esse que perdura por mais de 30 anos. No presente relatório, é apresentado um ECR multicêntrico, com moderada qualidade de evidência, recentemente publicado e com um número expressivo de pacientes, considerando-se a raridade da doença e que trouxe resultados significativamente superiores, quando comparado ao tratamento padrão isolado em praticamente todos os desfechos avaliados, além de perfil de seguração comparável ao tratamento atual. Na avaliação econômica, a adição de eltrombopague ao tratamento padrão trouxe vantagens clínicas e econômicas, mostrando ser custo-efetiva. Assim também, na análise de impacto orçamentário, a ampliação de seu uso, considerando-se que a tecnologia já está incorporada no SUS para o tratamento de TPI, trouxe economia de recursos em todos os cenários avaliados. RECOMENDAÇÃO PRELIMINAR DA CONITEC: No dia 09 de março de 2022, em sua 106ª Reunião Ordinária, os membros da Conitec deliberaram que a matéria fosse disponibilizada em consulta pública com recomendação preliminar favorável à ampliação de uso do eltrombopague para o tratamento adicional a imunossupressor em pacientes adultos com anemia aplástica grave. Considerou-se que as evidências apresentadas demonstraram eficácia e segurança acerca do tratamento proposto frente às alternativas terapêuticas já disponíveis no SUS, além de ser custo-efetivo e apresentar economia de recursos para o SUS. CONSULTA PÚBLICA: Foi realizada no período de 04/04/2022 a 25/04/2022. Foram recebidas 51 contribuições, sendo 21 pelo formulário técnico-científico e 30 pelo formulário sobre experiência ou opinião. Todas as contribuições recebidas concordaram com a recomendação preliminar da Conitec, sendo favoráveis à ampliação de uso da tecnologia. Assim, o entendimento da Conitec não foi alterado. RECOMENDAÇÃO FINAL DA CONITEC: Diante do exposto, os membros do Plenário da Conitec, em sua 108ª Reunião Ordinária, no dia 05 de maio de 2022, deliberaram por unanimidade recomendar a ampliação de uso do eltrombopague para o tratamento adicional a imunossupressor em pacientes adultos com anemia aplástica grave. Por fim, foi assinado o Registro de Deliberação nº 728/2022. DECISÃO: Ampliar o uso do eltrombopague para o tratamento adicional a imunossupressor em pacientes adultos com anemia aplástica grave, no âmbito do Sistema Único de Saúde - SUS conforme a Portaria nº 47, publicada no Diário Oficial da União nº 105, seção 1, página 78, em 3 de junho de 2022.


Subject(s)
Humans , Receptors, Thrombopoietin/agonists , Immunosuppressive Agents/therapeutic use , Anemia, Aplastic/drug therapy , Unified Health System , Brazil , Cost-Benefit Analysis/economics
7.
Fisioter. Bras ; 23(1): 73-79, Fev 11, 2022.
Article in Portuguese | LILACS | ID: biblio-1358411

ABSTRACT

Introdução: A anemia aplásica (AA) é uma condição clínica considerada rara que se desenvolve a partir da disfunção hematopoiética da medula óssea. O tratamento indicado é o transplante de células tronco hematopoiéticas (TCHP). Objetivo: Descrever o caso clínico e as estratégias utilizadas pela fisioterapia durante o processo de reabilitação física. Métodos: Trata-se de estudo de caso, realizado com paciente do sexo feminino, 34 anos de idade e diagnóstico de AA severa. Após avaliação clínica foi indicado o TCHP alogênico de um doador aparentado que apresentou compatibilidade histo-imunológica. O tempo total de internação hospitalar foi de 35 dias. Os objetivos da reabilitação física foram o de manter a ventilação pulmonar, prevenir o acúmulo de secreção, minimizar a progressão da fadiga, perda de força e resistência muscular. Resultados: A estratégia utilizada para contornar a extrema fragilidade hematológica e as implicações clínicas decorrentes evitou perda expressiva no desempenho no teste de caminhada de seis minutos (-10%) ao final da internação. Conclusão: Foi um verdadeiro desafio a implementação da reabilitação física durante o TCHP para o tratamento da AA, mas a estratégia adotada demonstrou-se segura, bem tolerada e suficiente para evitar maiores prejuízos no estado funcional. (AU)


Subject(s)
Female , Adult , Therapeutics , Bone Marrow , Physical Therapy Modalities , Health Strategies , Cell Transplantation , Fatigue , Walk Test , Frailty , Anemia, Aplastic
8.
Chinese Journal of Contemporary Pediatrics ; (12): 303-308, 2022.
Article in English | WPRIM | ID: wpr-928604

ABSTRACT

OBJECTIVES@#To study the association between paroxysmal nocturnal hemoglobinuria (PNH) clone and immunosuppressive therapy (IST) in children with severe aplastic anemia (SAA).@*METHODS@#A retrospective analysis was performed on the medical data of 151 children with SAA who were admitted and received IST from January 2012 to May 2020. According to the status of PNH clone, these children were divided into a negative PNH clone group (n=135) and a positive PNH clone group (n=16). Propensity score matching was used to balance the confounding factors, and the impact of PNH clone on the therapeutic effect of IST was analyzed.@*RESULTS@#The children with positive PNH clone accounted for 10.6% (16/151), and the median granulocyte clone size was 1.8%. The children with positive PNH clone had an older age and a higher reticulocyte count at diagnosis (P<0.05). After propensity score matching, there were no significant differences in baseline features between the negative PNH clone and positive PNH clone groups (P>0.05). The positive PNH clone group had a significantly lower overall response rate than the negative PNH clone group at 6, 12, and 24 months after IST (P<0.05). The evolution of PNH clone was heterogeneous after IST, and the children with PNH clone showed an increase in the 3-year cumulative incidence rate of aplastic anemia-PNH syndrome (P<0.05).@*CONCLUSIONS@#SAA children with positive PNH clone at diagnosis tend to have poor response to IST and are more likely to develop aplastic anemia-PNH syndrome.


Subject(s)
Child , Humans , Anemia, Aplastic/drug therapy , Clone Cells , Hemoglobinuria, Paroxysmal/etiology , Immunosuppression Therapy , Retrospective Studies
9.
Chinese Journal of Hematology ; (12): 300-304, 2022.
Article in Chinese | WPRIM | ID: wpr-929639

ABSTRACT

Objective: To study the metabolic characteristics of anti-human T-cell porcine immunoglobulin (p-ATG) in patients with severe aplastic anemia (SAA) . Methods: For patients with SAA treated with p-ATG combined cyclosporine A (CsA) immunosuppressants between February 2017 and December 2017, the p-ATG dose was 20 mg·kg(-1)·d(-1) over 12 h of intravenous administration for 5 consecutive days. The blood concentration of p-ATG was detected by the three-antibody sandwich ELISA method, the pharmacokinetic analysis software was fitted, and the second-chamber model method was used to calculate the pharmacokinetic parameters and plot the pharmacokinetic curve. Adverse events were recorded and the hematologic reactions were determined at 6 months after treatment. Results: Sixteen patients with SAA treated with p-ATG were enrolled, including 8 females and 8 males, with a median age of 22 years (range, 12 to 49 years) and a median weight of 62.5 kg (range, 37.5 to 82.0 kg) . The pharmacokinetics of p-ATG could be evaluated in 14 cases. p-ATG is distributed in vivo as a two-chamber model, with an average drug concentration peak (T(max)) of (5.786±2.486) days, a peak concentration (C(max)) of (616±452) mg/L, and a half-life of (10.479±8.242) days. The area under the drug time curve (AUC) was (5.807±3.236) mg/L·d. Six months after treatment, 8 of 14 patients received a hematologic response; the AUC (0-t) of the effective group and ineffective groups was (7.50±3.26) mg/L·d vs (4.50±2.18) mg/L·d, and the C(max) was (627±476) mg/L vs (584±382) mg/L, respectively. Conclusion: The plasma concentration of p-ATG reached a peak after 5 days of continuous infusion, and then decreased slowly, with a half-life of 10.479 days, and the residual drug concentration was detected in the body 60 days after administration. A relationship between drug metabolism and efficacy and adverse reactions could not be determined.


Subject(s)
Animals , Female , Humans , Male , Anemia, Aplastic/drug therapy , Antilymphocyte Serum/therapeutic use , Cyclosporine/therapeutic use , Immunoglobulins/therapeutic use , Immunosuppression Therapy , Immunosuppressive Agents/therapeutic use , Swine , T-Lymphocytes , Treatment Outcome
10.
Chinese Journal of Hematology ; (12): 393-399, 2022.
Article in Chinese | WPRIM | ID: wpr-929574

ABSTRACT

Objective: To reassess the predictors for response at 6 months in patients with severe or very severe aplastic anemia (SAA/VSAA) who failed to respond to immunosuppressive therapy (IST) at 3 months. Methods: We retrospectively analyzed the clinical data of 173 patients with SAA/VSAA from 2017 to 2018 who received IST and were classified as nonresponders at 3 months. Univariate and multivariate logistic regression analysis were used to evaluate factors that could predict the response at 6 months. Results: Univariate analysis showed that the 3-month hemoglobin (HGB) level (P=0.017) , platelet (PLT) level (P=0.005) , absolute reticulocyte count (ARC) (P<0.001) , trough cyclosporine concentration (CsA-C0) (P=0.042) , soluble transferrin receptor (sTfR) level (P=0.003) , improved value of reticulocyte count (ARC(△)) (P<0.001) , and improved value of soluble transferrin receptor (sTfR(△)) level (P<0.001) were related to the 6-month response. The results of the multivariate analysis showed that the PLT level (P=0.020) and ARC(△) (P<0.001) were independent prognostic factors for response at 6 months. If the ARC(△) was less than 6.9×10(9)/L, the 6-month hematological response rate was low, regardless of the patient's PLT count. Survival analysis showed that both the 3-year overall survival (OS) [ (80.1±3.9) % vs (97.6±2.6) %, P=0.002] and 3-year event-free survival (EFS) [ (31.4±4.5) % vs (86.5±5.3) %, P<0.001] of the nonresponders at 6 months were significantly lower than those of the response group. Conclusion: Residual hematopoietic indicators at 3 months after IST are prognostic parameters. The improved value of the reticulocyte count could reflect whether the bone marrow hematopoiesis is recovering and the degree of recovery. A second treatment could be performed sooner for patients with a very low ARC(△).


Subject(s)
Humans , Anemia, Aplastic/drug therapy , Antilymphocyte Serum/therapeutic use , Cyclosporine/therapeutic use , Immunosuppression Therapy , Immunosuppressive Agents/therapeutic use , Prognosis , Receptors, Transferrin/therapeutic use , Retrospective Studies , Treatment Outcome
11.
Chinese journal of integrative medicine ; (12): 20-27, 2022.
Article in English | WPRIM | ID: wpr-928930

ABSTRACT

OBJECTIVE@#To evaluate the efficacy and safety of Pai-Neng-Da Capsule (, panaxadiol saponins component, PNDC) in combination with the cyclosporine and androgen for patients with chronic aplastic anemia (CAA).@*METHODS@#A total of 79 CAA patients was randomly divided into 2 groups by a random number table, including PCA group [43 cases, orally PNDC 320 mg/d plus cyclosporine 5 mg/(kg·d) plus andriol 80 mg/d] and CA group [36 cases, orally cyclosporine 5 mg/(kg·d) plus andriol 160 mg/d]. All patients were treated and followed-up for 6 treatment courses over 24 weeks. The complete blood counts, score of Chinese medical (CM) symptoms were assessed and urine routine, electrocardiogram, hepatic and renal function were observed for safety evaluation. Female masculinization rating scale was established according to the actual clinical manifestations to evaluate the accurate degree of masculinization in female CAA patients treated by andriol.@*RESULTS@#The effective rates were 88.1% (37/42) in the PCA group and 77.8% (28/36) in the CA group based on the standard for the therapeutic efficacy evaluation of hematopathy. There was no significant difference in the white blood cell (WBC) counts, platelet counts and hemoglobin concentration of peripheral blood between two groups after 6 months treatment. The masculinization score of female patient in the PCA group was significantly lower than the CA group (P<0.05). The mild abdominal distention was observed in 1 cases in the PCA group. In CA group, the abnormalities in the hepatic function developed in 2 cases and the renal disfunction was found in 1 case.@*CONCLUSION@#The PNDC possesses certain curative effects in the treatment of CAA without obvious side-effects and can partially replace andriol thereby to reduce the degree of masculinization [Registried at Chinese Clinical Trial Registry (ChicTR1900028153)].


Subject(s)
Female , Humans , Androgens , Anemia, Aplastic/drug therapy , China , Nonprescription Drugs , Saponins/therapeutic use
12.
Journal of Experimental Hematology ; (6): 227-231, 2022.
Article in Chinese | WPRIM | ID: wpr-928698

ABSTRACT

OBJECTIVE@#To evaluate the clinical effect of haploid allogeneic hematopoietic stem cell transplantation(haplo-HSCT) in the treatment of severe aplastic anemia (SAA), and to explore the efficacy different between post-transplant cyclophosphamide (PT/Cy) and standard-dose ATG.@*METHODS@#The clinical data of 38 patients with SAA in our hospital from January 2012 to December 2019 were collected and retrospectively analyzed. The efficacy was evaluated. The patients with haplo-HSCT were divided into low-dose ATG combined with PT/Cy group and standard-dose ATG group, and the blood cell hematopoietic reconstruction time, GVHD incidence, mortality and survival time of the patients in the two groups was compared.@*RESULTS@#Among the 32 patients, hematopoietic reconstitution were detected in 9375%(30/32) recipients. The median time of neutrophil and platelet engraftment was 15(10-22) days and 13(7-30) days, respectively. The incidence of GVHD was 21.89%, the incidence of infection was 93.75%, and the 2-year overall survival rate was 84.38%. The hematopoietic reconstitution time, incidence of GVHD, mortality rate and survival time were no statistical differences between the patients in the two groups(all P>0.05).@*CONCLUSION@#Haplo-HSCT is an effective method for the treatment of SAA,low-dose ATG combined with PT/Cy can lighten the economic burden on patients, it would be a feasible treatment plan for SAA with light side effect.


Subject(s)
Humans , Anemia, Aplastic/therapy , Cyclophosphamide , Graft vs Host Disease , Haploidy , Hematopoietic Stem Cell Transplantation , Retrospective Studies , Transplantation Conditioning
13.
Journal of Experimental Hematology ; (6): 222-226, 2022.
Article in Chinese | WPRIM | ID: wpr-928697

ABSTRACT

OBJECTIVE@#To establish a stable mouse model of acquired aplastic anemia.@*METHODS@#Female BALB/C mice aged 6 months were intraperitoneally injected with cyclophosphamide and cyclosporine for 14 days. The number of peripheral blood cells, the concentration of hemoglobin, the number of bone marrow nucleated cells, bone marrow smear, bone marrow pathological sections and other indexes were observed.@*RESULTS@#In BALB/C mice injected intraperitoneally with cyclophosphamide and cyclosporine, the number of peripheral blood cells and the concentration of hemoglobin were significantly decreased, especially the white blood cells and platelets. Bone marrow smear showed a significant decrease in the number of nucleated cells and bone marrow hyperplasia. Bone marrow pathology showed decreased hematopoietic cells and increased non-hematopoietic cells such as adipocytes.@*CONCLUSION@#The mouse model with intraperitoneal injection of cyclophosphamide and cyclosporine can meet the diagnostic criteria of acquired aplastic anemia, which can be used as a mouse model for the study of the pathogenesis and treatment of acquired aplastic anemia.


Subject(s)
Animals , Female , Mice , Anemia, Aplastic , Bone Marrow , Cyclophosphamide , Cyclosporine , Mice, Inbred BALB C
14.
Frontiers of Medicine ; (4): 459-466, 2022.
Article in English | WPRIM | ID: wpr-939879

ABSTRACT

Whether Fanconi anemia (FA) heterozygotes are predisposed to bone marrow failure and hematologic neoplasm is a crucial but unsettled issue in cancer prevention and family consulting. We retrospectively analyzed rare possibly significant variations (PSVs) in the five most obligated FA genes, BRCA2, FANCA, FANCC, FANCD2, and FANCG, in 788 patients with aplastic anemia (AA) and hematologic malignancy. Sixty-eight variants were identified in 66 patients (8.38%). FANCA was the most frequently mutated gene (n = 29), followed by BRCA2 (n = 20). Compared with that of the ExAC East Asian dataset, the overall frequency of rare PSVs was higher in our cohort (P = 0.016). BRCA2 PSVs showed higher frequency in acute lymphocytic leukemia (P = 0.038), and FANCA PSVs were significantly enriched in AA and AML subgroups (P = 0.020; P = 0.008). FA-PSV-positive MDS/AML patients had a higher tumor mutation burden, higher rate of cytogenetic abnormalities, less epigenetic regulation, and fewer spliceosome gene mutations than those of FA-PSV-negative MDS/AML patients (P = 0.024, P = 0.029, P = 0.024, and P = 0.013). The overall PSV enrichment in our cohort suggests that heterozygous mutations of FA genes contribute to hematopoietic failure and leukemogenesis.


Subject(s)
Humans , Anemia, Aplastic/genetics , Epigenesis, Genetic , Fanconi Anemia/genetics , Germ Cells , Hematologic Neoplasms/genetics , Leukemia, Myeloid, Acute/genetics , Retrospective Studies
15.
Journal of Experimental Hematology ; (6): 824-831, 2022.
Article in Chinese | WPRIM | ID: wpr-939695

ABSTRACT

OBJECTIVE@#To explore the improvement effect of CXC chemokine receptor 4 (Cxcr4) gene-modified bone marrow mesenchymal stem cell (BMSC)-derived exosomes on aplastic anemia (AA), and make a preliminary exploration of the mechanism.@*METHODS@#Mouse BMSCs were isolated and cultured, then infected by recombinant lentivirus carrying Cxcr4 gene. The expression of green fluorescence was observed through fluorescence microscope, the expression of Cxcr4 mRNA was detected by real-time fluorescence quantitative PCR, and the BMSC-derived exosomes modified with Cxcr4 gene were extracted. Mouse models of AA were constructed, and control group, model group (AA), AA+BMSC group, AA+NC-BMSC group, AA+Cxcr4-BMSC group were set up. Except control group and model group, the other three groups of mice were injected 400 μl exosomes from different sources via the tail vein, after 2 weeks, the routine blood indices and the number of bone marrow nucleated cells were detected, the pathological changes of bone marrow were observed by HE staining, and the expression level of Treg cells was detected by flow cytometry.@*RESULTS@#Mouse BMSCs were successfully isolated, and BMSCs with high expression of Cxcr4 and their exosomes were obtained. Compared with the control group, the number of red blood cell (RBC), white blood cell (WBC), and platelet (PLT), the hemoglobin (Hb) content and proportion of Treg cells in the peripheral blood of mice in the model group significantly decreased (P<0.01), as well as the number of bone marrow nucleated cells (P<0.01). The proliferation level of nucleated cells was low, and the medullary cavity was filled with a large number of fat cells. Compared with the model group, the number of RBC, WBC, PLT, the Hb content and proportion of Treg cells in the peripheral blood of mice in the AA+BMSC group, AA+NC-BMSC group, and AA+Cxcr4-BMSC group significantly increased (P<0.01), as well as the number of bone marrow nucleated cells (P<0.01), and pathological changes of bone marrow were improved. In addition, the number of RBC, WBC, PLT, the Hb content and proportion of Treg cells in the peripheral blood of mice in the AA+Cxcr4-BMSC group were significantly higher than those in the AA+BMSC group (P<0.01), as well as the number of bone marrow nucleated cells (P<0.01).@*CONCLUSION@#Injection of Cxcr4 gene-modified BMSC-derived exosomes has a certain improvement effect on AA mice, and the mechanism may be related to an increase of the proportion of Treg cells.


Subject(s)
Animals , Humans , Mice , Anemia, Aplastic/metabolism , Bone Marrow Cells , Exosomes/metabolism , Mesenchymal Stem Cells , Receptors, CXCR4
16.
Journal of Experimental Hematology ; (6): 819-823, 2022.
Article in Chinese | WPRIM | ID: wpr-939694

ABSTRACT

OBJECTIVE@#To observe the occurrence of immune dysfunction in children with aplastic anemia (AA) and the factors that may lead to immune dysfunction, analyze the relationship between the expression of granulocyte colony stimulating factor (G-CSF) and immune dysfunction.@*METHODS@#A total of 34 children with AA treated in our hospital from December 2016 to September 2018 were selected. All the children received immunosuppressive therapy (IST) for 6 months. According to whether the children had immune dysfunction after 6 months of treatment, they were divided into occurrence group and non occurrence group. General information and laboratory indices were compared between the two groups, and serum G-CSF level was tested, the relationship between serum G-CSF level and immune dysfunction in AA children after treatment with IST was observed and analyzed.@*RESULTS@#After treatment with IST for 6 months, 12 cases developed immune dysfunction (35.29%). Serum interferon (IFN)-γ level of the occurrence group was higher but G-CSF level was lower than those of the non occurrence group (P<0.05), while the difference of other baseline data was not statistically significant (P>0.05). Multiple regression analysis showed that overexpression of serum IFN-γ and low expression of G-CSF were both the influencing factors of immune dysfunction in AA children after IST treatment (OR>1, P<0.05). ROC curve was drawn, and the result showed that the area under the curve (AUC) of serum G-CSF level predicted the risk of immune dysfunction after IST was 0.843>0.80, when the index cut-off value was set at 6.614 pg/ml, the predictive value was ideal.@*CONCLUSION@#AA children have a higher risk of immune dysfunction after IST, which may be related to the low expression of serum G-CSF. The detection of serum G-CSF expression can be considered to predict the risk of immune dysfunction in AA children after IST, so as to guide early clinical intervention.


Subject(s)
Child , Humans , Anemia, Aplastic , Antilymphocyte Serum/therapeutic use , Cyclosporine/therapeutic use , Granulocyte Colony-Stimulating Factor , Immunity , Immunosuppressive Agents/therapeutic use
17.
Afr. j. lab. med. (Print) ; 11(1): 1-7, 2022. tables
Article in English | AIM | ID: biblio-1379111

ABSTRACT

Background: Paroxysmal nocturnal haemoglobinuria (PNH) clones in children are rare but commonly associated with aplastic anaemia (AA) and myelodysplasia.Objective: This study aimed to determine the prevalence of PNH clones in paediatric patients with idiopathic AA, identify differences in clinical and laboratory features and outcomes, and determine the impact of clone size on clinical presentation.Methods: Patients with confirmed idiopathic AA who were tested for PNH between September 2013 and January 2018 at the Inkosi Albert Luthuli Central Hospital, Durban, KwaZulu-Natal, South Africa, were included. PNH clones were detected in neutrophils and monocytes by flow cytometry using fluorescent aerolysin, CD24, CD66b and CD14. Results: Twenty-nine children with AA were identified and 11 were excluded. Ten patients (10/18, 55.6%) had PNH clones ranging from 0.11% to 24%. Compared to the PNH-negative group, these children were older (median: 10 years vs 4 years, p= 0.02) and had significantly lower total white cell counts (median 1.7 × 109/L vs 3.2 × 109/L; p= 0.04). There was no difference in median absolute neutrophil count or haemoglobin concentration. Four patients in each group received immunosuppressive therapy (IST). At six months, all four patients with PNH clones had responded, compared to one in the PNH-negative group. Conclusion: More than half of children with AA had a PNH clone. The size of the clone did not impact clinical severity; however, IST use may positively impact prognosis. We recommend early initiation of IST in patients with AA to avoid delays associated with human leukocyte antigen typing.


Subject(s)
Humans , Male , Female , Integrative Pediatrics , Anemia, Aplastic , Histocompatibility Testing , Dyspnea, Paroxysmal , Flow Cytometry
18.
African Health Sciences ; 22(3): 81-92, 2022-10-26. Tables
Article in English | AIM | ID: biblio-1401048

ABSTRACT

Background: Anaemia in pregnancy is a known public health problem in South Africa. Maternal, perinatal morbidity and mortality are known to be associated with anaemia in pregnancy. Very little is known from literature with regards to the progression of anaemia during the antenatal period of pregnancy. Objectives: To estimate haemoglobin levels, the prevalence and determinants of anaemia at different gestational ages and to show the trend. Method: A retrospective cohort (follow-up) study was conducted using the antenatal clinic register. Prevalence rates of anaemia (haemoglobin < 11 g/dl) at different gestational ages were measured. Factors associated with anaemia were assessed using chisquare test and stepwise multivariate logistic regression analysis. Results: A total of 801 pregnant women were enrolled at the booking visit and followed-up during their antenatal period. The prevalence of anaemia at the booking visit was 37%. The prevalence of anaemia at 20, 26, 32 and 36 weeks of gestation were 36.6%, 39.6%, 39.8% and 29.2% respectively. Binary logistic regression at the booking visit showed that teenage women were 2.5 times more likely to have anaemia (OR=2.5, p=0.005) than older women. Women who booked during the first trimester were 60% less likely to have anaemia (OR= 0.40, P=0.005) at the booking visit and 62% less likely to be anaemic at 36 weeks of gestation (OR=0.38, p=0.013) compared to those who booked late for antenatal care. Conclusion: Prevalence of anaemia during pregnancy was high. Early booking for antenatal care was a predictor for lower rate of anaemia. Thus, health education strategy should be encouraged for early antenatal booking


Subject(s)
Prenatal Care , Hemoglobins , Pregnancy , Anger Management Therapy , Anemia, Aplastic , South Africa , Prevalence
19.
African Health Sciences ; 22(3): 718-725, 2022-10-26. Figures, Tables
Article in English | AIM | ID: biblio-1401990

ABSTRACT

Background: Functional iron deficiency has been found to be a common cause of poor response to erythropoiesis stimulating agents in anaemic patients with chronic kidney disease (CKD). Objectives: Assess the functional iron status of patients with chronic kidney disease. Methods: This was a hospital based cross sectional study. The study subjects were chronic kidney disease patients with age and sex matched healthy controls. Full blood count, serum ferritin, soluble transferring receptor, C-reactive protein, serum iron and total iron binding capacity were measured in the patients and healthy controls. Data was analyzed with statistical package for the social sciences software version 22.0. And the level of statistical significance was set at p. value < 0.05. Results: The mean ± SD of the age of patient with CKD was 55.0 + 15.4 years, while that of controls was 52.7 + 13.6 years. The mean serum ferritin, serum iron, TIBC and CRP were significantly higher in patients compared with controls (p<0.001, 0.023, <0.001 and 0.001) respectively. Functional iron deficiency was seen in 19.5% of patients with CKD. Conclusion: The predominant form of iron deficiency in our study was functional iron deficiency


Subject(s)
Colonic Diseases, Functional , Renal Insufficiency, Chronic , Iron Deficiencies , Anemia, Aplastic , Patients , Hematinics , Nigeria
20.
Journal of Experimental Hematology ; (6): 944-950, 2021.
Article in Chinese | WPRIM | ID: wpr-880173

ABSTRACT

OBJECTIVE@#To investigate the clinical characteristics and risk factors of cytomegalovirus (CMV) infection after allogeneic hematopoietic stem cell transplantation (allo-HSCT) in patients with severe aplastic anemia (SAA).@*METHODS@#Clinical data from 270 SAA patients with allo-HSCT were retrospectively analyzed, including 108 sib congruence patients and 162 substitute donors (68 unrelated donor congruence patients and 94 related haploid patients). Different pretreatment schemes were selected for different transplantation modes. The HLA-identical sibling and haploid grafts were all bone marrow and peripheral blood stem cells, and the grafts from unrelated donors were peripheral blood stem cells. After granulocyte implantation, blood CMV-DNA was regularly monitored. Flow cytometry was also used to determine the absolute number of CD3@*RESULTS@#CMV infection occurred in 229 of 270 patients with an incidence of 84.8%. Among them, 18 patients developed giant cell disease. Univariate analysis showed that alternative donors (unrelated total and haploid donors), mycophenolate mofetil and acute graft-versus-host disease were statistically significantly associated with CMV infection (P<0.05). Multivariate analysis showed that alternative donors were associated with CMV infection. The recovery of CD3@*CONCLUSION@#After allo-HSCT, substitute donors are more easily to develop CMV infection than full-sibling donors, and the reconstruction of immune function is delayed after transplantation.


Subject(s)
Humans , Anemia, Aplastic , Cytomegalovirus Infections , Graft vs Host Disease , Hematopoietic Stem Cell Transplantation , Retrospective Studies
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