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1.
Pediatr Blood Cancer ; 43(3): 289-94, 2004 Sep.
Article in English | MEDLINE | ID: mdl-15266416

ABSTRACT

Here we report our combined analysis of vaginal mesonephric adenocarcinoma (MA) in a 13 years old treated at our institution and of reported vaginal or cervical MA and clear-cell adenocarcinoma in 37 patients 18 years old or younger. Tumors in 27 patients arose in the vagina; 8 (21%) had metastases at diagnosis. The predominant symptom was vaginal bleeding; 62% of patients were exposed to diethylstilbestrol in utero. The 3-year survival estimate was 71% +/- 11%. Surgical treatment and metastasis at diagnosis were significant predictors of survival. The association between tumor excision and excellent outcome suggests that radical hysterectomy or pelvic exenteration is unnecessary. Radiotherapy as an adjunct may have contributed to this outcome.


Subject(s)
Adenocarcinoma/pathology , Uterine Cervical Neoplasms/pathology , Vaginal Neoplasms/pathology , Adenocarcinoma/drug therapy , Adenocarcinoma/radiotherapy , Adenocarcinoma/surgery , Adolescent , Child , Child, Preschool , Female , Humans , Infant , Neoplasm Metastasis/pathology , Prognosis , Survival Rate , Time Factors , Treatment Outcome , Uterine Cervical Neoplasms/drug therapy , Uterine Cervical Neoplasms/radiotherapy , Uterine Cervical Neoplasms/surgery , Vaginal Neoplasms/drug therapy , Vaginal Neoplasms/radiotherapy , Vaginal Neoplasms/surgery
2.
Proc Natl Acad Sci U S A ; 99(13): 8932-7, 2002 Jun 25.
Article in English | MEDLINE | ID: mdl-12084934

ABSTRACT

Treatment with isolated allogeneic mesenchymal cells has the potential to enhance the therapeutic effects of conventional bone marrow transplantation in patients with genetic disorders affecting mesenchymal tissues, including bone, cartilage, and muscle. To demonstrate the feasibility of mesenchymal cell therapy and to gain insight into the transplant biology of these cells, we used gene-marked, donor marrow-derived mesenchymal cells to treat six children who had undergone standard bone marrow transplantation for severe osteogenesis imperfecta. Each child received two infusions of the allogeneic cells. Five of six patients showed engraftment in one or more sites, including bone, skin, and marrow stroma, and had an acceleration of growth velocity during the first 6 mo postinfusion. This improvement ranged from 60% to 94% (median, 70%) of the predicted median values for age- and sex-matched unaffected children, compared with 0% to 40% (median, 20%) over the 6 mo immediately preceding the infusions. There was no clinically significant toxicity except for an urticarial rash in one patient just after the second infusion. Failure to detect engraftment of cells expressing the neomycin phosphotransferase marker gene suggested the potential for immune attack against therapeutic cells expressing a foreign protein. Thus, allogeneic mesenchymal cells offer feasible posttransplantation therapy for osteogenesis imperfecta and likely other disorders originating in mesenchymal precursors.


Subject(s)
Bone Marrow Transplantation , Osteogenesis Imperfecta/therapy , Absorptiometry, Photon , Base Sequence , Cell Division , Child, Preschool , DNA Primers , Female , Humans , Male , Osteogenesis Imperfecta/immunology , Osteogenesis Imperfecta/pathology , Transplantation, Homologous
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