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3.
J Pediatr Hematol Oncol ; 34(2): 146-50, 2012 Mar.
Article in English | MEDLINE | ID: mdl-22009006

ABSTRACT

Plerixafor has been recently approved by the European Medicines Agency for adult patients who have failed other mobilization strategies. Experience in children, however, is extremely limited. We describe the experience of the use of this drug in 8 children under a compassionate-use program in 3 Italian and 2 Spanish centers. Plerixafor was generally well tolerated; only 2 of 8 children reported adverse effects, and these were mild in intensity. Peripheral blood progenitor cell priming was improved with plerixafor in 6 of 8 patients. In the remaining 2 patients, the target CD34+ cell count was below the target of 2 × 10(6) cells/kg, although in these patients cell counts before collection were good enough for leukapheresis. Plerixafor, therefore seems to be safe and effective for peripheral blood progenitor cell mobilization in children. Adverse events were comparable with those described with filgrastim alone.


Subject(s)
Granulocyte Colony-Stimulating Factor/therapeutic use , Hematopoietic Stem Cell Mobilization/methods , Hematopoietic Stem Cells/drug effects , Heterocyclic Compounds/therapeutic use , Adolescent , Benzylamines , Child , Compassionate Use Trials , Cyclams , Cytokines/therapeutic use , Female , Filgrastim , Hematologic Neoplasms/surgery , Hematopoietic Stem Cell Transplantation/methods , Humans , Male , Recombinant Proteins/therapeutic use , Retrospective Studies
4.
Appl Immunohistochem Mol Morphol ; 18(2): 150-8, 2010 Mar.
Article in English | MEDLINE | ID: mdl-19770707

ABSTRACT

Malignant rhabdoid tumors (MRTs) are aggressive childhood neoplasms, occurring mainly in the kidney and brain. We describe 2 unusual cases of extrarenal and noncranial location (liver and soft tissue with dissemination) mimicking hepatoblastoma, neuroblastoma or Ewing sarcoma. Both cases revealed a polyphenotypic profile, combined with cytokeratin, vimentin, and CD99 expression. INI1/BAF-47 showed negative protein nuclear expression in both cases, suggesting a diagnosis of MRT. An extensive immunohistochemical panel was performed to exclude pediatric tumors reminiscent of MRT. The genetic studies failed to detected MYCN amplification, 11q23 deletion, and EWS break-apart positivity. No alterations of 22q integrity were demonstrated with the probes used for the study (N25 Di George/22q11.2, 22qter, and EWS/22q12). We discuss the differential diagnosis in pediatric polyphenotypic tumors (Wilms tumor, neuroblastoma, desmoplastic small round cell tumor, and Ewing sarcoma). Analysis of INI1/BAF-47 expression can offer important clues in the diagnosis of pediatric tumors with rhabdoid phenotype. The integration of clinical, morphologic, immunohistochemical, and genetic data is required to approach a correct diagnosis of pediatric tumor in unusual location with atypical or undifferentiated morphology.


Subject(s)
Immunohistochemistry , Liver Neoplasms/diagnosis , Neoplasms, Multiple Primary/diagnosis , Rhabdoid Tumor/diagnosis , Skin Neoplasms/diagnosis , 12E7 Antigen , Antigens, CD , Antineoplastic Combined Chemotherapy Protocols/therapeutic use , Cell Adhesion Molecules , Chromosomal Proteins, Non-Histone/metabolism , Chromosome Aberrations , DNA-Binding Proteins/metabolism , Diagnosis, Differential , Drug Resistance, Neoplasm , Fatal Outcome , Female , Humans , Infant , Infant, Newborn , Keratins/metabolism , Liver Neoplasms/drug therapy , Liver Neoplasms/pathology , Liver Neoplasms/physiopathology , N-Myc Proto-Oncogene Protein , Neoplasms, Multiple Primary/drug therapy , Neoplasms, Multiple Primary/pathology , Neoplasms, Multiple Primary/physiopathology , Nuclear Proteins/genetics , Oncogene Proteins/genetics , RNA-Binding Protein EWS/genetics , Rhabdoid Tumor/drug therapy , Rhabdoid Tumor/physiopathology , Rhabdoid Tumor/secondary , SMARCB1 Protein , Skin Neoplasms/drug therapy , Skin Neoplasms/physiopathology , Skin Neoplasms/secondary , Transcription Factors/metabolism , Vimentin/metabolism
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