RESUMEN
Patients with hematologic malignancies not in remission before allogeneic hematopoietic stem cell transplantation (HSCT) have a poor prognosis. To improve the antitumor activity of conditioning, we combined clofarabine with myeloablative doses of busulfan in a phase 1/2 study in nonremission hematologic malignancies. Forty-six patients were enrolled, including 31 patients with nonremission acute myelogenous leukemia (AML). Patients had a median age of 53 years, with a median comorbidity index of 3. Donors were unrelated, HLA mismatched, or both in 59% of patients. Common grade III to IV nonhematologic toxicities included transient transaminitis (50%), mucositis (24%), hand-foot syndrome (13%), transient hypoxia (13%), nausea/vomiting (9%), and diarrhea (9%). All patients engrafted. Complete remission was achieved in 80% of all patients by day +30 and in 100% of AML patients without prior hematopoietic stem cell transplantation. Two-year nonrelapse mortality for all patients was 31%, and overall survival was 28%. In AML, the overall survival was 48% at 1 year and 35% at 2 years. These data suggest that clofarabine combined with myeloablative doses of busulfan is well tolerated, secures engraftment, and possesses significant antitumor activity, particularly in nonremission AML. This study is registered at www.ClinicalTrials.gov under identifier NCT00556452.
Asunto(s)
Nucleótidos de Adenina/administración & dosificación , Antineoplásicos/administración & dosificación , Arabinonucleósidos/administración & dosificación , Busulfano/administración & dosificación , Supervivencia de Injerto/efectos de los fármacos , Trasplante de Células Madre Hematopoyéticas , Leucemia/mortalidad , Leucemia/terapia , Agonistas Mieloablativos/administración & dosificación , Acondicionamiento Pretrasplante/métodos , Nucleótidos de Adenina/efectos adversos , Adolescente , Adulto , Anciano , Antineoplásicos/efectos adversos , Arabinonucleósidos/efectos adversos , Busulfano/efectos adversos , Preescolar , Clofarabina , Supervivencia sin Enfermedad , Humanos , Lactante , Masculino , Persona de Mediana Edad , Agonistas Mieloablativos/efectos adversos , Inducción de Remisión , Tasa de Supervivencia , Factores de Tiempo , Acondicionamiento Pretrasplante/efectos adversos , Trasplante HomólogoRESUMEN
Idiopathic pulmonary hemosiderosis (IPH) is a rare and life-threatening disorder. Early diagnosis and appropriate management are essential for their better prognosis and patients' quality of life (QOL). It is considered that Down syndrome patients with IPH have a worse prognosis compared to other IPH cases. A 2-year-old girl with Down syndrome received the diagnosis of IPH after two episodes of massive pulmonary hemorrhage requiring assist ventilation, who suffered from recurrent IPH during tapering period of oral corticosteroid, started liposteroid therapy. We report here a case of successful control of recurrent IPH and improved QOL enormously with tapering dose of corticosteroid after starting liposteroid therapy.