rome and Thalassemia

Bone marrow transplantation (BMT) for class 3 patients with thalassemia is challenging due to high rates of graft rejection and transplant-related mortality. Since the first studies of BMT in the late 1980s, a number of conditioning regimens have been designed to improve outcomes, but with suboptimal results. Here we report the outcome of transplantation in class 3 patients using a modified protocol.. Sixty-three patients between 5 and 16.7 years of age with class 3 thalassemia received HLA-matched sibling BMT following either the original protocol (26 patients) or the modified protocol (37 patients). Both regimens comprised preconditioning cytoreduction with hydroxyurea and azathioprine starting at -45 days pretransplant, and fludarabine from days -16 to -12. Conditioning was performed with busulfan and cyclophosphamide (original protocol) or with busulfan, thiotepa, and cyclophosphamide (modified protocol).. The 2 groups showed similar patient demographics. At day 0, the degree of cytoreduction (lymphopenia, neuthropenia, and thrombocytopenia) achieved by the modified protocol was greater than the original protocol. The incidence of graft failure/rejection was significantly higher in the original group (15%; 95% confidence interval [95% CI], 5-32%) compared with the modified group (0%) (P = 0.014). The respective 5-year thalassemia-free survival rates were 73% (95% CI, 51-86%) and 92% (95% CI, 77-97%) (P = 0.047). Both groups showed similar incidences of grades II to IV acute graft-versus host disease. Modified protocol did not increase nonhematological toxicity or infectious complications.. The modified treatment protocol effectively and safely prevented graft failure/rejection and significantly increased thalassemia-free survival of class 3 patients with thalassemia.

Topics: Adolescent; Age Factors; Bone Marrow Transplantation; Child; Child, Preschool; Disease-Free Survival; Drug Therapy, Combination; Female; Graft Rejection; Graft Survival; Graft vs Host Disease; Histocompatibility; Histocompatibility Testing; HLA Antigens; Humans; Immunosuppressive Agents; Incidence; Kaplan-Meier Estimate; Living Donors; Male; Predictive Value of Tests; Prospective Studies; Risk Factors; Rome; Siblings; Thalassemia; Time Factors; Transplantation Conditioning; Treatment Outcome

Using an immunological method (immunoblot), we have established that hemoglobin (or hemoglobin fragments) can be quantitatively determined in old and ancient bones, some of them dating back 4500 years. It is shown that the total recovery decreases with time, but it is still effective in the older specimens. Thus, the immunological assay may prove useful to solve problems relevant to paleontology and paleopathology.

Topics: Antibodies, Anti-Idiotypic; Hemoglobinometry; Hemoglobins; Hemoglobins, Abnormal; History, Ancient; Humans; Italy; Lumbar Vertebrae; Paleontology; Paleopathology; Rome; Thalassemia

Topics: Female; Fetal Blood; Genetic Carrier Screening; Humans; Pregnancy; Pregnancy Complications, Hematologic; Prenatal Diagnosis; Rome; Thalassemia

Extensive population surveys begun in Italy in 1943 revealed a high incidence of mycrocytemia. Health authorities established a national organization to combat microcytemia which financed and directed activities throughout Italy from 1955 to 1971. The work performed by the microcytemia centers in screening and prophylaxis is described. The results of a screening program underway since 1967 involving 46,559 students of the University of Rome are presented in detail. A preliminary survey of 256 families revealed that the incidence of subsequent births after the births of a child affected by Cooley's anemia was generally low. Results obtained to date are encouraging and justify more extensive programs for the prevention and treatment of microcytemia.

Topics: Adolescent; Child; Child, Preschool; Community Health Services; Erythrocytes; Female; Genetic Counseling; Health Education; Hemoglobins; Humans; Italy; Male; Mass Screening; Osmotic Fragility; Rome; Thalassemia