Challenges and opportunities for HSCT outcome registries: perspective from international HSCT registries experts.

Patient registries, frequently referred to as outcome registries, are 'organized systems' that use observational study methods to collect uniform data. Registries are used to evaluate specified outcomes for a population defined by a particular disease, condition or exposure that serves one or more predetermined scientific, clinical or policy purposes. Outcome registries were established very early in the development of hematopoietic SCT (HSCT).

Related and unrelated nonmyeloablative hematopoietic stem cell transplantation for malignant diseases.

Patients with advanced hematological malignancies ineligible for conventional myeloablative allogeneic hematopoietic stem cell transplantation (HSCT) due to advanced age or medical contraindications were enrolled in multi-center study to investigate the safety and efficacy of nonmyeloablative HSCT using a 2 Gy total body irradi ation (TBI)-based regimen. A total of 192 patients (median age 55) were treated with HLA-matched sibling peripheral blood stem cell (PBSC) grafts, and 63 patients (median age 53) received a 10 of 10 HLA-antigen matched unrelated donor (URD) HSCT (PBSC graft, n = 48; marrow graft, n = 15). Diagnoses included multiple myeloma (n = 61), myelodysplastic syndrome (n = 55), chronic myeloid leukemia (n = 31), non-Hodgkin lymphoma (n = 31), acute myeloid leukemia (n = 28), chronic lymphocytic leukemia (n = 24), Hodgkin Disease (n = 14).

Double high-dose chemotherapy with autologous peripheral stem cell rescue in relapsed Wilms' tumor.

We performed double high-dose chemotherapy (HDCT) and autologous stem cell rescue (ASCR) on an 8-year-old girl after relapse of Wilms' tumor with unfavorable histology. Relapse occurred while the patient was still on first-line therapy and showed multiple adverse prognostic features. Since depleted hematopoietic reserves restricted further conventional therapy, a double HDCT approach was chosen to achieve maximal dose intensity.