Treatment of Diamond Blackfan Anemia With Antithymocyte Globulin and Cyclosporine A
Antithymocyte globulin
+ Cyclosporine
Troubles de l'insuffisance de la moelle osseuse+13
+ Syndromes Congénitaux d'Insuffisance Médullaire
+ Anémie
Étude thérapeutique
Résumé
Date de début de l'étude : 1 juillet 1998
Date à laquelle le premier participant a commencé l'étude.Diamond Blackfan anemia (DBA) is a constitutional pure red cell aplasia of unknown etiology. There is laboratory evidence for an immune mechanism and most patients respond to corticosteroids. However the relapse and failure rate are high, and corticosteroids are associated with many short and long term side effects. Patients who do not respond or who do not tolerate corticosteriods require lifelong red blood cell transfusion and iron chelation therapy. Allogeneic bone marrow transplantation is an option for those with a related histocompatible donor, but this procedure is associated with high mortality and morbidity. Other therapies have been tried without general success. Occasional responses to either ATG or cyclosporine have been reported, but no study has used both ATG and cyclosporine. In other blood/bone marrow disorders of immune etiology these drugs have synergistic effects. We propose a Phase II study to explore the combined use of ATG and cyclosporine as a rational approach to the treatment of Diamond Blackfan anemia.
Protocole
Cette section fournit des détails sur le plan de l'étude, y compris la manière dont l'étude est conçue et ce qu'elle évalue.25 participants à inclure
Nombre total de participants que l'essai clinique vise à recruter.Traitement
Éligibilité
Les chercheurs recherchent des patients correspondant à une certaine description appelée critères d'éligibilité : état de santé général ou traitements antérieurs du patient.Tout sexe
Le sexe biologique des participants éligibles à s'inscrire.Volontaires sains non autorisés
Indique si les individus en bonne santé et ne présentant pas la condition étudiée peuvent participer.Conditions
Pathologie
Critères
INCLUSION CRITERIA: Diagnosis of DBA as characterized by a hyporegenerative anemia presenting in early childhood with reticulocytopenia, and low or absent erythroid precursors in the bone marrow. Transfusion-dependence due to steroid failure or intolerance of steroid side effects. Ineligible for or declining an allogeneic transplant. Ages 3 to 75. EXCLUSION CRITERIA: Serum creatinine greater than 2 times normal or a creatinine clearance less than 50% normal. SGPT or SGOT greater than 5 times normal. History of epilepsy (any seizures besides childhood febrile seizures). Current pregnancy or unwillingness to take oral contraceptives if menstruating. Positive diepoxybutane (DEB) test for Fanconi anemia. HIV positivity. Inability or unwillingness to sign an informed consent, either by the patient, or in the case of a minor, by the parent or guardian responsible for the patient. Underlying organ failure and/or those with a Karnofsky performance status of less than 1. Treatment with androgens, prednisone greater than 10 mg/day, growth factors, or other immunosuppressive therapies within one month of protocol entry. Ongoing treatment with Beta-adrenergic blocking drugs. Previous treatment with ATG and concurrent CSA. Previous treatment with either drug alone is acceptable if greater than one year prior to study entry.
Centres d'étude
Ce sont les hôpitaux, cliniques ou centres de recherche où l'essai est conduit. Vous pouvez trouver le site le plus proche de vous ainsi que son statut.Cette étude comporte 1 site
National Heart, Lung and Blood Institute (NHLBI)
Bethesda, United StatesOuvrir National Heart, Lung and Blood Institute (NHLBI) dans Google Maps