RESUMO
BACKGROUND: While second-generation tyrosine kinase inhibitors (TKI) revolutionized treatment for patients with chronic myeloid leukemia (CML) who developed a suboptimal response to imatinib, many patients in developing countries are fixed to the latter due to socioeconomic barriers. Despite this scenario, scarce information is available to evaluate the clinical prognosis of these patients. METHODS: We conducted a retrospective cohort analysis to compare the overall mortality of patients with CML who developed a suboptimal response to a standard dose of imatinib and were treated with either high-dose imatinib or a second-generation TKI. We created a marginal structural model with inverse probability weighting and stabilized weights. Our primary outcome was overall survival (OS) at 150 months. Our secondary outcomes were disease-free survival (DFS) at 150 months and adverse events. RESULTS: The cohort included 148 patients, of which 32 received high-dose imatinib and 116 a second-generation TKI. No difference was found in the 150-month overall survival risk (RR: 95% CI 0.91, 0.55-1.95, P-value = .77; RD: -0.04, -0.3 to 0.21, P-value = .78) and disease-free survival (RR: 1.02, 95% CI 0.53-2.71, P-value = .96; RD: 0.01, -0.26 to 0.22, P-value = .96). There was also no difference in the incidence of adverse events in either group. CONCLUSION: Ideally, patients who develop a suboptimal response to imatinib should be switched to a second-generation TKI. If impossible, however, our findings suggest that patients treated with high-dose imatinib have a similar overall survival and disease-free survival prognosis to patients receiving a second-generation TKI.
Assuntos
Mesilato de Imatinib , Leucemia Mielogênica Crônica BCR-ABL Positiva , Humanos , Hispânico ou Latino , Mesilato de Imatinib/administração & dosagem , Leucemia Mielogênica Crônica BCR-ABL Positiva/tratamento farmacológico , Leucemia Mielogênica Crônica BCR-ABL Positiva/mortalidade , Estudos Retrospectivos , Substituição de MedicamentosRESUMO
La micosis fungoides y el síndrome de Sezary están constituidos por un grupo de linfomas no Hodgkin indolentes, extranodales, con origen en linfocitos T; afectan la piel de manera primaria; son generalmente incurables y los pacientes tienen mal pronóstico porque la enfermedad es habitualmente refractaria a diversas modalidades terapéuticas. El trasplante de médula ósea alogénico es una opción terapéutica más. La alta mortalidad del acondicionamiento pretrasplante convencional ha hecho que se considere el trasplante alogénico con acondicionamiento de intensidad reducida como una opción viable para esta enfermedad. Se presenta el caso de una paciente con micosis fungoides a quien se hizo un trasplante hematopoyético con un esquema no mieloablativo y en quien se logró remisión completa sostenida de la enfermedad.
Sezary syndrome (SS) and mycosis fungoides (MF) are a group of non Hodgkin lymphomas that originate from T-lymphocytes and involve mostly the skin. These entities are generally non treatable and patient prognosis remains poor even with the advent of current treatment schedules. Complete remissions are seldom observed. For this reason, bone marrow transplant has been used as a treatment option. The high mortality associated with this procedure has turned reduced intensity conditioning stem cell transplant into a treatment option. This case study illustrates how stem cell transplant offers complete remission of this type of lymphomas.
Assuntos
Humanos , Feminino , Adulto , Transplante de Células-Tronco Hematopoéticas , Micose Fungoide/cirurgia , Neoplasias Cutâneas/cirurgia , Indução de RemissãoRESUMO
En los últimos ocho años se han llevado a cabo, en diversas partes del país y en otros países en desarrollo, trasplantes de células hematopoyéticas alogénicas usando el Método Mexicano de acondicionamiento no ablativo que se desarrolló en nuestro país con base en otros métodos de acondicionamiento de intensidad reducida empleados en otras partes del mundo. Habiendo trasplantado ya más de 350 pacientes, se han hecho análisis de los resultados de acuerdo con los padecimientos que han motivado la práctica de los injertos. Los mejores resultados se han obtenido en pacientes con leucemia granulocítica crónica e hipoplasia medular, en tanto que los menos halagüeños se han obtenido en individuos con leucemia aguda linfoblástica, con resultados intermedios en pacientes trasplantados por leucemia aguda mieloblástica. La práctica de los trasplantes hematopoyéticos empleando el Método Mexicano, además de haber beneficiado a varios pacientes quienes no habrían podido trasplantarse, ha incidido en el incremento de la actividad académica relacionada con los trasplantes en el país.
In the past eight years, in Mexico and in other developing countries, over 350 patients have undergone allogeneic hematopoietic stem cell transplants using a non-myeloablative conditioning regimen developed in Mexico and based on international standards. The so called [quot ]Mexican method[quot ] to conduct allogeneic stem cell transplants is endowed with certain advantages which make it affordable and in turn, available to individuals living in resource-poor countries. The best results using this method have been observed among patients with stage 1 chronic myelogenous leukemia and aplastic anemia. The less favourable results have been observed among patients with acute lymphoblastic leukemia; mild to moderate results have been reported among patients with acute myelogenous leukemia. The [quot ]Mexican method[quot ] to conduct hematopoietic cells allografting has resulted not only in turning this method accessible to patients in developing countries, but also it has witnessed an increase in the academic activities of physicians from these countries involved in the field.
Assuntos
Humanos , Leucemia/cirurgia , Transplante de Células-Tronco Hematopoéticas/métodos , Leucemia/mortalidade , México , Taxa de Sobrevida , Fatores de Tempo , Transplante HomólogoRESUMO
In two institutions in México, twelve patients were given a second allogeneic stem cell transplantation, using the "Mexican" non-myeloablative preparative regimen. Eight had a malignant condition (six acute leukemias, one myelofibrosis and one myelodysplasia), eleven individuals were allografted twice from the same donor and in one case, cells from two different umbilical cords were used. The median time to conduct the second allograft after the first one was 6 months (range 1-41). The five patients who failed to engraft after the first transplant failed also to engraft after the second one; all of them had been heavily transfused. Only three patients were successfully rescued with the second transplant, two with acute leukemia and one with aplastic anemia. Seven patients are alive 10-41 months (median 35) after the second transplant, but only three (25%) remain disease-free. The 52-month overall survival (SV) of the patients is 58%, whereas the median overall SV has not been reached, being above 52 months. Conducting a second allograft may be useful to rescue some individuals relapsing after a first hematopoietic allotransplant.
Assuntos
Transplante de Células-Tronco de Sangue do Cordão Umbilical , Transplante de Células-Tronco de Sangue Periférico , Condicionamento Pré-Transplante/métodos , Doença Aguda , Adulto , Anemia Aplástica/cirurgia , Pré-Escolar , Estudos de Coortes , Transplante de Células-Tronco de Sangue do Cordão Umbilical/estatística & dados numéricos , Intervalo Livre de Doença , Feminino , Custos de Cuidados de Saúde , Hospitais/estatística & dados numéricos , Hospitais Universitários/estatística & dados numéricos , Humanos , Lactente , Leucemia/cirurgia , Masculino , México , Pessoa de Meia-Idade , Defeitos do Tubo Neural/cirurgia , Osteopetrose/cirurgia , Transplante de Células-Tronco de Sangue Periférico/estatística & dados numéricos , Mielofibrose Primária/cirurgia , Recidiva , Aplasia Pura de Série Vermelha/cirurgia , Reoperação/estatística & dados numéricos , Análise de Sobrevida , Talassemia/cirurgia , Transplante Homólogo/estatística & dados numéricos , Resultado do TratamentoRESUMO
In two institutions in México, twelve patients were given a second allogeneic stem cell transplantation, using the "Mexican" non-myeloablative preparative regimen. Eight had a malignant condition (six acute leukemias, one myelofibrosis and one myelodysplasia), eleven individuals were allografted twice from the same donor and in one case, cells from two different umbilical cords were used. The median time to conduct the second allograft after the first one was 6 months (range 1-41). The five patients who failed to engraft after the first transplant failed also to engraft after the second one; all of them had been heavily transfused. Only three patients were successfully rescued with the second transplant, two with acute leukemia and one with aplastic anemia. Seven patients are alive 10-41 months (median 35) after the second transplant, but only three (25%) remain disease-free. The 52-month overall survival (SV) of the patients is 58%, whereas the median overall SV has not been reached, being above 52 months. Conducting a second allograft may be useful to rescue some individuals relapsing after a first hematopoietic allotransplant.
En dos instituciones en México se llevaron a cabo doce segundos trasplantes de células hematopoyéticas usando el "método mexicano" de acondicionamiento no mieloablativo. Ocho pacientes tenían una enfermedad maligna (seis leucemias agudas, una mielofibrosis y una mielodisplasia). Once sujetos se retrasplantaron del mismo donador y en un caso se emplearon células hematopoyéticas de dos diferentes cordones umbilicales. La mediana del tiempo transcurrido entre los dos trasplantes fue de seis meses (rango 1 a 41). Los cinco pacientes que no se injertaron con el primer trasplante tampoco se injertaron con el segundo; todos ellos habían sido multitransfundidos antes de los trasplantes. Sólo tres pacientes se pudieron rescatar con el segundo trasplante, dos con leucemia aguda y uno con anemia aplástica. Siete pacientes están vivos 10 a 41 meses (mediana 35) después del segundo trasplante, pero sólo tres (25%) se encuentran libres de enfermedad. La supervivencia (SV) global a 52 meses es de 58%, en tanto que la mediana de SV no se ha alcanzado y es mayor de 52 meses. Hacer un segundo trasplante hematopoyético puede rescatar a algunos pacientes quienes recaen después de un trasplante de médula ósea.
Assuntos
Adulto , Pré-Escolar , Feminino , Humanos , Lactente , Masculino , Pessoa de Meia-Idade , Transplante de Células-Tronco de Sangue do Cordão Umbilical , Transplante de Células-Tronco de Sangue Periférico , Condicionamento Pré-Transplante/métodos , Doença Aguda , Anemia Aplástica/cirurgia , Estudos de Coortes , Transplante de Células-Tronco de Sangue do Cordão Umbilical/estatística & dados numéricos , Intervalo Livre de Doença , Custos de Cuidados de Saúde , Hospitais Universitários/estatística & dados numéricos , Hospitais/estatística & dados numéricos , Leucemia/cirurgia , México , Defeitos do Tubo Neural/cirurgia , Osteopetrose/cirurgia , Transplante de Células-Tronco de Sangue Periférico/estatística & dados numéricos , Mielofibrose Primária/cirurgia , Recidiva , Aplasia Pura de Série Vermelha/cirurgia , Reoperação/estatística & dados numéricos , Análise de Sobrevida , Resultado do Tratamento , Talassemia/cirurgia , Transplante Homólogo/estatística & dados numéricosRESUMO
A group of 21 consecutive patients aged 4-20 (median 13) years was prospectively allografted using a reduced intensity preparative regimen. The group included both malignant (acute lymphoblastic leukemia, acute myelogenous leukemia, and chronic myelogenous leukemia) and nonmalignant (aplastic anemia, Diamond-Blackfan anemia, thalassemia major and adrenoleukodystrophy) conditions. Follow-up times ranged between 16 and 1038 days. Four of 21 patients (9.5%) developed acute graft-versus-host disease, and 2 of them died, whereas limited chronic graft-versus-host disease was observed in 2 of 15 cases. The 100-day mortality was 19%. Median overall survival was above 1038 days, whereas the 34-month survival was 55%. These data show that reduced intensity stem cell transplantation in children permits rapid engraftment from siblings with little toxicity.