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1.
Brasília; CONITEC; maio 2022.
Non-conventional in Portuguese | LILACS, ColecionaSUS, BRISA | 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
2.
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)
Androgens , Anemia, Aplastic/drug therapy , China , Female , Humans , Nonprescription Drugs , Saponins/therapeutic use
3.
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)
Anemia, Aplastic/drug therapy , Child , Clone Cells , Hemoglobinuria, Paroxysmal/etiology , Humans , Immunosuppression Therapy , Retrospective Studies
4.
Article in Chinese | WPRIM | ID: wpr-921532

ABSTRACT

Objective To compare the efficacy and safety of cyclosporin A(CsA)and CsA combined with recombined human erythropoietin(rhEPO)in the treatment of patients with chronic aplastic anemia(CAA).Methods Data of 79 patients with CAA treated at Department of Hematology,PUMC Hospital between January 2016 and June 2018 were collected for retrospective analysis.Forty-five patients were treated with CsA+rhEPO,and the other 34 patients with CsA alone.All the enrolled patients were treated for at least 1.5-2.0 years and followed for at least 1.0 year.The efficacy,side effects,long-term outcomes were compared between the two groups,and factors that may influence the efficacy were analyzed.Results The patients treated with CsA+rhEPO included 14 males and 31 females,with a median age of 43(19,73)years old.The median treatment duration of CsA and rhEPO was 26(12,38)and 4(3,6)months,respectively,and the median followed-up time was 24(12,42)months.The patients treated with CsA alone included 16 males and 18 females,with a median age of 36(16,85)years old.The median CsA treatment duration was 24(12,40)months and the median follow-up time was 25(12,40)months.There was no statistical difference in baseline characteristics between the two groups(all


Subject(s)
Adolescent , Adult , Aged , Aged, 80 and over , Anemia, Aplastic/drug therapy , Cyclosporine/therapeutic use , Erythropoietin/therapeutic use , Female , Humans , Male , Middle Aged , Recombinant Proteins , Remission Induction , Retrospective Studies , Young Adult
5.
Journal of Experimental Hematology ; (6): 1257-1261, 2021.
Article in Chinese | WPRIM | ID: wpr-888548

ABSTRACT

OBJECTIVE@#To summarize the long-term efficacy of cyclosporine (CsA) in the treatment of non-severe aplastic anemia (NSAA) in children, and explore the early significant indicators.@*METHODS@#Data of 36 NSAA children in Department of Hematological Oncology, Wuhan Children's Hospital, Tongji Medical College of Huazhong University of Science and Technology from January 2013 to December 2017 were analyzed retrospectively. All the children received oral CsA immunosuppressive therapy, and CsA trough concentration was checked to maintain at the rage of 200-250 μg/L after 2 weeks. The evaluation time points were at 3, 6, 12, 18 and 24 months, and assessment items were peripheral white blood cell differential count and reticulocyte's percentage and count.@*RESULTS@#The 36 NSAA cases were composed of 16 males and 20 females, whose median age was 5.46 (2.92-7.99) years old, and median follow-up time was 28.00 (10.00-38.25) months. After taking oral CsA for 24 months, the number of cumulative effective cases was 21. There were 4 cases of complete remission (CR), 17 cases of partial remission (PR), and 15 cases of non-remission (NR). The total effective rate was 58.33%, and median effect-acting time of CsA was 3.0 (0.5-10.0) months. Compared with ineffective group, neutrophil (NEU) and red blood cell (RBC) of effective group (CR+PR) began to increase significantly at the 3rd month, and hemoglobin (Hb), platelet (PLT) and white blood cell (WBC) increase significantly at the 6th month after oral CsA administration (P<0.05). Except for 2 cases who received component transfusion within 3-12 months after taking oral CsA for 3 months in effective group, the others did not need.@*CONCLUSION@#The overall effective rate of oral CsA in children with NSAA was 58.33%. Stopping blood transfusion after the 3 months of treatment may be considered as a turning point for disease outcomes, and levels of NEU, RBC at the 3rd month and Hb, PLT, WBC at the 6th month as indicators for predicting disease prognosis.


Subject(s)
Anemia, Aplastic/drug therapy , Child , Child, Preschool , Cyclosporine , Female , Humans , Immunosuppressive Agents , Male , Prognosis , Retrospective Studies , Treatment Outcome
9.
Indian J Pediatr ; 2008 Mar; 75(3): 229-33
Article in English | IMSEAR | ID: sea-78680

ABSTRACT

OBJECTIVE: To assess the responses to ATG and cyclosporin combination in patients of aplastic anemia. METHODS: Twenty three (17M: 6F) patients of aplastic anemia (11 very severe aplastic anemia (VSAA) and 12 severe aplastic anemia (SAA), were administered antithymocyte globulin and cyclosporin. RESULTS: The median age of patents was 8 years (range 6-12 years). Three patients died within 2 months of therapy. Twenty children (11 SAA and 9 VSAA) were finally analysed. Six months after the start of treatment, 8/20 (40%) patients responded-2 complete (CR) and 6 partial responses (PR). At the end of 1 year; 2 patients maintained CR and seven patients continued PR (overall responders 45%). The response was better in SAA (54.5%) with 2 CR and 4 PR; than in VSAA (33%) with 3 PR . Eleven (55%) children were alive without response. One patient developed AML 13 months later. CONCLUSION: We conclude that antithymocyte globulin and cyclosporin combination is an effective treatment for aplastic anemia patients who are ineligible for bone marrow transplantation.


Subject(s)
Anemia, Aplastic/drug therapy , Antilymphocyte Serum/therapeutic use , Child , Cyclosporine/therapeutic use , Female , Humans , Immunosuppressive Agents/therapeutic use , Male , Recurrence , Treatment Outcome
10.
Iranian Journal of Allergy, Asthma and Immunology. 2006; 5 (1): 39-40
in English | IMEMR | ID: emr-164206

ABSTRACT

Polyclonal anti-thymocyte globulin [ATG] is used as an immunosuppressive agent in the treatment of aplastic anemia [AA]. Serum sickness is a recognized side effect of ATG. We observed abnormal skin manifestation in patient with aplastic anemia who had been treated with ATG. We conclude that abnormal immune function caused by aplastic anemia and ATG and corticosteroids may aggravate the signs of serum sickness


Subject(s)
Humans , Male , Anemia, Aplastic/drug therapy , Serum Sickness/pathology , Skin Diseases/chemically induced , Serum Sickness/etiology
11.
Säo Paulo med. j ; 122(6): 273-275, Nov. 4, 2004. graf
Article in English | LILACS | ID: lil-393198

ABSTRACT

CONTEXTO: O prognóstico da anemia aplástica grave melhorou com o advento do transplante de medula óssea e do tratamento imunossupressor com globulina antitimocitária. Em contraste com o sucesso destes protocolos, os estudos com seguimento a longo prazo mostraram a ocorrência de doenças clonais, tais como: hemoglobinúria paroxística noturna, síndrome mielodisplásica e leucemia aguda. RELATO DE CASO: Nós relatamos o primeiro caso descrito no Brasil de um paciente com anemia aplástica que evoluiu para síndrome mielodisplásica e leucemia mielóide aguda associada a presença de hemoglobina H e aumento da hemoglobina fetal.


Subject(s)
Humans , Male , Adult , Anemia, Aplastic/complications , Hemoglobin H , Leukemia, Myeloid/etiology , Acute Disease , Anemia, Aplastic/drug therapy , Anemia, Aplastic/surgery , Antilymphocyte Serum/administration & dosage , Antilymphocyte Serum/adverse effects , Antineoplastic Combined Chemotherapy Protocols/adverse effects , Bone Marrow Transplantation/adverse effects , Fatal Outcome , Globins/biosynthesis , Leukemia, Myeloid/drug therapy , Myelodysplastic Syndromes/complications , Time Factors
12.
Rev. méd. Chile ; 132(8): 989-994, ago. 2004. tab
Article in Spanish | LILACS | ID: lil-384196

ABSTRACT

A 28 years old male on chronic hemodialysis for 40 months due to a IgA crescentic glomerulonephritis developed pancytopenia (hematocrit 16 percent, white blood cell count 3.800 mm3 and platelets 11.000 mm3. The bone marrow aspirate showed erythropoietic hyperplasia. Hemolytic anemia, folate or vitamin B12 deficiency and paroxysmal nocturnal hemoglobinuria were ruled out. Steroids were given with a transient elevation of red cells and platelets, which lasted only for some weeks. Afterwards, intravenous immunoglobulin was given without benefit. Two months after, a bone marrow biopsy and a bone marrow magnetic resonance imaging showed severe aplasia. Cyclosporine was started with a rapid increase in blood cells count. Eight months later, he received a renal transplant from a cadaveric donor. Immunosupression was achieved with cyclosporine, prednisone and mycofenolate mofetil. The patient required hemodialysis for the first three weeks and a mild acute cellular rejection was treated with methylprednisolone. At discharge, 6 weeks later, serum creatinine was 2.4 mg/dl and creatinine clearance 37.6 ml/min. During the first months after transplant, platelet count and hemoglobin decreased and a bone marrow biopsy showed only mild hypoplasia. Four months after renal transplant the hematocrit was 43 percent, white blood cell count 6.600 mm3 and platelets, 150.000 mm3 and did not change during the first year of follow up (Rev Méd Chile 2004; 132: 989-94).


Subject(s)
Adult , Humans , Female , Immunosuppressive Agents/therapeutic use , Anemia, Aplastic/etiology , Anemia, Aplastic/drug therapy , Cyclosporine/therapeutic use , Renal Dialysis/adverse effects , Glomerulonephritis, IGA/therapy , Kidney Transplantation , Pancytopenia/therapy
13.
Rev. méd. Chile ; 131(12): 1439-1443, dic. 2003. ilus
Article in Spanish | LILACS | ID: lil-360243

ABSTRACT

Severe aplastic anemia has an elevated mortality if treatment is unsatisfactory. Immunosuppression is the treatment of choice in adults, comparable with allogeneic bone marrow transplant in children. We report two adult patients (both males, aged 59 and 67 years old) who were treated successfully with lymphoglobulin and cyclosporine. The initial response started within 3 months of treatment and was almost complete after 2 years, when cyclosporine was stopped. After three years, both patients have almost normal blood counts, with minor sequels: avascular necrosis of both femoral heads due to the use steroids, that recovered spontaneously in 1 patient and reduced vision due to thrombocytopenic retinal hemorrhages, in the other (Rev Méd Chile 2003; 131: 1439-43).


Subject(s)
Humans , Male , Middle Aged , Anemia, Aplastic/drug therapy , Antilymphocyte Serum/therapeutic use , Cyclosporins/therapeutic use , Immunosuppressive Agents/therapeutic use , Anemia, Aplastic/etiology , Bone Marrow/pathology , Combined Modality Therapy
14.
Rev. chil. pediatr ; 74(2): 179-185, mar.-abr. 2003. tab
Article in Spanish | LILACS | ID: lil-348477

ABSTRACT

La anemia aplástica (AA) es una falla medular caracterizada por pancitopenia en sangre periférica como resultado de una disminución de la producción de células sanguíneas en médula ósea. Tiene diversas etiologías y una incidencia de 2 a 4 casos por 1 000 000 niños menores de 15 años. El tratamiento de elección es el transplante de médula ósea alogénico o en su defecto la inmunosupresión con lingoglobulina o timoglobulina además de ciclosporina, metilprednisolona y factores de crecimiento hematopoyético. Se presenta la experiencia con 7 pacientes del Hospital Roberto del Río diagnosticados entre los años 1995 y 2000, edad 2 meses a 13 años, con biopsia de médula ósea compatible. Dos pacientes presentaban etiología congénita, 3 con antecedentes de hepatitis y 2 fueron considerados idiopáticos. Un paciente fue transplantado de un hermano compatible luego de recibir inmunosupresión, 3 recibieron inmunosupresión con linfo/timoglobulina además de ciclosporina y factor estimulante de colonias de granulocitos y 3 niños sólo recibieron tratamiento de sostén con metilprednisolona o factores de crecimiento. Dos pacientes fallecieron precozmente por cuadro infeccioso. Cinco pacientes están vivos con una mediana de seguimiento de 43 meses, los 4 que recibieron inmunosupresión incluido el paciente transplantado, y la paciente con anemia de Fanconi


Subject(s)
Humans , Male , Adolescent , Child , Female , Anemia, Aplastic/drug therapy , Cyclosporine , Anemia, Aplastic/surgery , Hematopoietic Cell Growth Factors/pharmacology , Immunosuppression Therapy , Methylprednisolone , Pancytopenia , Receptors, Colony-Stimulating Factor , Transplantation, Homologous
15.
Article in English | WPRIM | ID: wpr-199786

ABSTRACT

Aplastic anemia is a rare complication of thymoma and is extremely infrequent after thymectomy. We present a case of a 60-year-old woman with very severe aplastic anemia appearing sixteen months after thymectomy for a thymoma. She underwent thymectomy for a thymoma in April 2000. Preoperative examination revealed no hematologic abnormality. About sixteen months after the operation, she was readmitted because of pancytopenia with cough and fever. Bone marrow aspiration revealed a very severe hypoplasia in all the three cell lines with over 80% fatty tissue, and chest CT revealed no recurrence of thymoma. Her aplastic anemia had responded to cyclosporine A and granulocyte-colony stimulating factor (G-CSF).


Subject(s)
Anemia, Aplastic/drug therapy , Biopsy, Needle , Bone Marrow/pathology , Cyclosporine/administration & dosage , Drug Therapy, Combination , Female , Follow-Up Studies , Granulocyte Colony-Stimulating Factor/administration & dosage , Humans , Middle Aged , Rare Diseases , Risk Assessment , Severity of Illness Index , Thymectomy/adverse effects , Thymoma/diagnosis , Treatment Outcome
16.
Article in English | IMSEAR | ID: sea-87363

ABSTRACT

OBJECTIVE: The present study was conducted to evaluate the efficacy of low dose cyclosporine-A in the patients of severe aplastic anaemia, who cannot afford allogenic bone marrow transplantation and immunosuppressive therapy with antithymocyte globulin. METHODS: The diagnosis of severe aplastic anaemia was established by standard criteria. Twelve patients were given cyclosporine-A orally at a dose of 6 mg/kg body weight in divided doses in two daily equal proportions for six months. Eleven patients were put on oral stanozolol in the dosage of 1 mg/kg body weight/day in divided doses. All surviving patients were evaluated at three and six months. RESULTS: At the end of six months, 41.66% of twelve patients responded to cyclosporine-A. One patient had complete response and four patients had partial response. Only one out of 11 patients receiving stanozolol responded. CONCLUSIONS: i) Cyclosporine-A is a viable therapeutic option in the treatment of severe aplastic anaemia, ii) Low dose cyclosporine-A is able to slow down the stormy course of the severe aplastic anaemia, iii) Androgens have very little effect on the survival of patients of severe aplastic anaemia.


Subject(s)
Administration, Oral , Adult , Anabolic Agents/administration & dosage , Anemia, Aplastic/drug therapy , Child , Cyclosporine/administration & dosage , Female , Humans , Immunosuppressive Agents/administration & dosage , Male , Prognosis , Stanozolol/administration & dosage , Time Factors
17.
Article in English | WPRIM | ID: wpr-127195

ABSTRACT

To compare magnetic resonance (MR) images of the bone marrow (BM) after bone marrow transplantation or immunosuppressive therapy in patients with aplastic anemia (AA), MR imaging of BM was reviewed retrospectively in 16 patients (13 males and 3 females, mean age 26 yr) with AA who completely responded clinically after transplantation or immunosuppressive therapy. The signal intensity (SI) of BM was classified into four patterns according to the increasing amount of cellular marrow, i.e., pattern I to IV. SI of MR imaging of BM exhibited an increase of cellular marrows following both transplantation and immunosuppressive therapy. Of the eight patients on transplantation, the SI of the lumbar spinal BM was pattern III in two patients and IV in six on T1-weighted and short tau inversion recovery (STIR) images. In the eight patients with immunosuppressive therapy, the SI of the lumbar spinal BM was pattern II in one, III in five, and IV in two on T1-weighted images and pattern II in one, III in four, and IV in three on STIR images. SI on MR imaging of the lumbar spinal BM showed a more cellular pattern in patients on transplantation than in those on immunosuppressive therapy.


Subject(s)
Adult , Anemia, Aplastic/drug therapy , Bone Marrow Transplantation , Comparative Study , Female , Humans , Immunosuppressive Agents/therapeutic use , Lumbar Vertebrae , Magnetic Resonance Imaging , Male , Pelvis , Retrospective Studies
18.
Braz. j. med. biol. res ; 33(5): 553-8, May 2000. graf
Article in English | LILACS | ID: lil-260250

ABSTRACT

Severe aplastic anemia (SAA) is probably an immune-mediated disorder, and immunosuppressive therapy is recommended for patients with no available donor for bone marrow transplant. Between October 1984 and November 1987, 25 consecutive children and adolescents with SAA with no HLA-compatible marrow donor received equine antithymocyte globulin (ATG) (15 mg kg-1 day-1) for 10 days. The patients were evaluated 6 weeks, 6 months, and 12 months after starting ATG treatment. Thereafter, patients were evaluated yearly until July 1998. Median age was 10 years (range, 1.5-20 years), granulocyte counts on referral ranged from 0.032 to 1.4 x 10(9)/l (median 0.256 x 10(9)/l), and 12 patients had granulocyte counts < 0.2 x 10(9)/l. At a median follow-up of 9.6 years (range, 8.6-11.8 years), 10 patients (40 percent) remained alive with good marrow function. No morphologic evidence of hematological clonal disorders has been observed, although two patients probably have acquired clonal chromosomal abnormalities (trisomy 8 and del(6)q21, respectively). Responses to ATG were observed between 6 weeks and 6 months from the start of treatment in 60 percent of evaluable patients. The response rate was not different in patients whose granulocyte count at diagnosis was < 0.2 x 10(9)/l, or in those who were < 10 years of age. This study supports the view that, when compared with supportive measures, ATG is an effective treatment for children or adolescents with SAA. Although these results are inferior to those reported for marrow transplantation or more intensive immunosuppressive regimens, these patients who responded to ATG are long-term survivors with stable peripheral blood counts and a low rate of relapse.


Subject(s)
Humans , Animals , Child , Child, Preschool , Infant , Adolescent , Adult , Anemia, Aplastic/drug therapy , Antilymphocyte Serum/therapeutic use , Immunosuppressive Agents/therapeutic use , Anemia, Aplastic/mortality , Antilymphocyte Serum/adverse effects , Bone Marrow/physiopathology , Cell Count , Cohort Studies , Follow-Up Studies , Granulocytes , Immunosuppressive Agents/adverse effects , Recurrence , Survival Rate , Treatment Outcome
20.
Rev. chil. infectol ; 16(2): 87-93, 1999. tab
Article in Spanish | LILACS | ID: lil-257957

ABSTRACT

Desde su descubrimiento hace menos de 25 años, mucho se ha progresado en el conocimiento de la infección causada por parvovirus B19, reconociéndose actualmente un amplio espectro de manifestaciones y la existencia de infecciones crónicas o persistentes. Este agente debe ser considerado en el diagnóstico diferencial de las variadas entidades clínicas que se describen en esta revisión. En este artículo se discuten también aspectos históricos, virológicos, epidemiológicos, de diagnóstico y manejo terapéutico de las infecciones por parvovirus B19


Subject(s)
Humans , Erythema Infectiosum/etiology , Parvoviridae Infections/etiology , Parvovirus B19, Human/pathogenicity , Anemia, Aplastic/etiology , Anemia, Aplastic/drug therapy , Anti-Inflammatory Agents, Non-Steroidal/therapeutic use , Erythema Infectiosum/diagnosis , Erythema Infectiosum/drug therapy , Erythema Infectiosum/transmission , Immunocompromised Host , Hydrops Fetalis/drug therapy , Hydrops Fetalis/etiology , Immunoglobulins, Intravenous/therapeutic use , Parvoviridae Infections/diagnosis , Parvovirus B19, Human/drug effects , Parvovirus B19, Human/isolation & purification
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