New drugs for childhood anemia.
New drugs, recently available for treatment of different forms of anaemia, have somehow changed the therapeutic scenario in paediatric haematology. The aim of this review is to focus on the newest molecules discussing indications, clinical usefulness and related problems. Erythropoietin, the specific growth factor of red cell precursors, is now an established option for anaemia of chronic renal failure, prematurity, bone marrow transplantation and chemotherapy. Anti-CD20 monoclonal antibody, a novel cytotoxic molecule for mature B lymphocytes, has proven to be effective in the treatment of refractory autoimmune cytopenias. Haemoglobin analogues are currently under investigation, in order to obtain a synthetic oxygen-carrier that can substitute blood transfusions. Finally drugs that are able to increase the production of haemoglobin F have been used in thalassemias and haemoglobinopathies. For patients with sickle cell disease, hydroxyurea is no longer an experimental tool; it has given rise to several trials, where it has proven to be effective in terms of both clinical and haematological improvement.
La Spina
M
M
Regional Center of Hematology and Pediatric Oncology, University of Catania, Catania, Italy. milenalaspina@virgilio.it
Russo
G
G
D000328:Adult; D000367:Age Factors; D000740:Anemia / Q000139:chemically induced / Q000188:drug therapy* / Q000209:etiology; D000744:Anemia, Hemolytic, Autoimmune / Q000188:drug therapy; D000755:Anemia, Sickle Cell / Q000188:drug therapy; D000911:Antibodies, Monoclonal / Q000008:administration & dosage / Q000627:therapeutic use; D058846:Antibodies, Monoclonal, Murine-Derived; D000970:Antineoplastic Agents / Q000009:adverse effects; D000986:Antisickling Agents / Q000627:therapeutic use; D001802:Blood Substitutes / Q000008:administration & dosage / Q000627:therapeutic use; D016026:Bone Marrow Transplantation; D002087:Butyrates / Q000008:administration & dosage / Q000627:therapeutic use; D002648:Child; D002986:Clinical Trials as Topic; D004921:Erythropoietin / Q000008:administration & dosage / Q000627:therapeutic use; D005319:Fetal Hemoglobin / Q000032:analysis; D005544:Forecasting; D006453:Hemoglobinopathies / Q000188:drug therapy; D006454:Hemoglobins; D006801:Humans; D006918:Hydroxyurea / Q000008:administration & dosage / Q000627:therapeutic use; D007231:Infant, Newborn; D007235:Infant, Premature, Diseases / Q000188:drug therapy*; D007676:Kidney Failure, Chronic / Q000150:complications; D016377:Organ Transplantation; D000069283:Rituximab; D013789:Thalassemia / Q000188:drug therapy; D014019:Tissue Donors
D000911:Antibodies, Monoclonal; D058846:Antibodies, Monoclonal, Murine-Derived; D000970:Antineoplastic Agents; D000986:Antisickling Agents; D001802:Blood Substitutes; D002087:Butyrates; D006454:Hemoglobins; D004921:Erythropoietin; C096798:HBOC 201; D000069283:Rituximab; D005319:Fetal Hemoglobin; D006918:Hydroxyurea