No abstract available.

Gaucher's disease (GD) is the most common inherited lysosomal storage disease, manifested by generalized accumulation of glucocerebroside in macrophages of the reticuloendothelial system due to a deficient lysosomal beta-glucocerebrosidase (GC). It is inherited by an autosomal recessive pattern in which three clinical phenotypes have been described based on the presence and severity of neurologic involvement. GD is treated possible by GC enzyme replacement therapy, allogeneic bone marrow transplantation (BMT), and gene therapy. We here report the exprience of successful allogeneic BMT in a 16-year-old female patient with GD type III which was demostrated markedly increased Gaucher cells in bone marrow and absence of GC activity in peripheral blood monocytes by FACS using 5'- pentafluorobenzoylaminofluorescein-di-beta-D-glucoside (PFBFDGlu) as substrate. Donor marrow engraftment was confirmed by chromosome analysis using microsatellite and by bone marrow examination. Assay of GC activity using FACS revealed normal level of enzyme activity. She remains alive and well after 12 months of BMT.
Adolescent ; Bone Marrow Examination ; Bone Marrow Transplantation* ; Bone Marrow* ; Enzyme Replacement Therapy ; Female ; Gaucher Disease* ; Genetic Therapy ; Glucosylceramidase ; Humans ; Lysosomal Storage Diseases ; Macrophages ; Microsatellite Repeats ; Monocytes ; Mononuclear Phagocyte System ; Phenotype ; Tissue Donors

PURPOSE: Tbis phase II study was performed to evaluate the efficacy and safety of docetaxel in patients with anthracycline-pretreated metastatic breast cancer (MBC). MATERIALS AND METHODS: From September 1996 to January 1998, 27 patients with metastatic breast cancer from 31 to 63 years of age with a performance status of 0 to 2 were registered in the phase II trial. All patients had metastatic breast cancer which had progressed or relapsed 2 during or after treatment with an anthracycline-based regimen. Docetaxel 75 mg/m2 was ad- ministered over 1 hour every 21 days until disease progression was documented or until toxic effects precluded further therapy. All patients received dexamethasone orally at the dose of 16 mg on days -1, 0, 1 of each cycle. RESULTS: Objective responses were seen in 9 of 25 assessable patients (two complete and seven partial responses), with an overall objective response rale of 36%. The median duration of response was 36 weeks (range 19.0~40.5). The median time to progression and survival duration were 17.5 and 69 weeks, respectively, for assessable patients. One hundred fifty cycles (median, five) of docetaxel were administered. Among 27 patients assessable for toxicity, the following side effects were reported: nadir neutropenia grade 3 (4 patients) and grade 4 (22 patients); grade 2 stomatitis (6 patients); grade 2 alopecia (5 patients); grade 2 to 3 neurosensory toxicity (4 patients); no hypersensitivity reaction; mild fluid retention (4 patients). CONCLUSION: Docetaxel is an active agent in patients with antracycline-pretreated metastatic breast cancer. Docetaxel was associated with severe but reversible neutropenia. Dexamethasone prevented hypersensitivity reactions and appeared to ameliorate fluid retention.
Alopecia ; Breast Neoplasms* ; Breast* ; Dexamethasone ; Disease Progression ; Humans ; Hypersensitivity ; Neoplasm Metastasis ; Neutropenia ; Respiratory Sounds ; Stomatitis

PURPOSE: Most malignant peritoneal or pleural effusions caused by advanced malignancy are unresponsive to systemic chemotherapy except for chemotherapy sensitive tumors, and they are equally ineffective to regional therapy or radiotherapy. Thus, for the purpose of palliating the symptoms related to malignant effusion and to reduce fluid reaccumulations, we evaluated the therapeutic feasibility and efficacy of intracavitary ' Ho-CHICO (chito- san complex) instillation for intractable malignant effusions. MATERIALS AND METHODS: Thirty one patients with cytologically or pathologically proven malignant effusions underwent intracavitary 166Ho-CHICO therapy from May 1996 to March 1998 at Ajou University Hospital. The subjective and objective responses were evaluated 4 weeks after the treatment, including the changes of symptoms, weight, abdominal girth, doses of diuretics, frequencies and amounts of repeat aspirations for fluid reaccumulations, and imaging studies of chest radiograph and ultrasounds. RESULTS: The response rates treated with Ho-CHICO were 50% in patients with peritoneal effusion and 46% in patients with pleural effusion (overall 49%). The response rates between 166Ho-CHICO doses of 50-80 mCi and 90-100 mCi were similar (50% vs 47%). Response rate of 70% was noted in patients with even distribution of radioisotope on the post-therapy scan, but, the response rate was lower in cases with focal (44%) and uneven (29%) distribution pattern. There was no difference in response by the effusion sites. All patients tolerated intracavitary 166Ho-CHICO instillation well, although the majority of patients experienced Grade I/II side effects such as pain, fever, weakness and dyspnea. But, no serious complications of Grade lII or IV degree were observed with 166Ho-CHICO therapy. CONCLUSION: Intracavitary 166Ho-CHICO instillation was clinically efficacious in controlling malignant effusions without a significant toxicity seen with conventional sclerotic therapy. The therapeutic modality appeared to offer similar benefits obtained with the conventional intracavitary therapy.
Ascitic Fluid ; Aspirations (Psychology) ; Chitosan* ; Diuretics ; Drug Therapy ; Dyspnea ; Fever ; Holmium* ; Humans ; Pleural Effusion* ; Radiography, Thoracic ; Radiotherapy ; Ultrasonography

Diphenylhydantoin (DPH) is one of the most widely used anticonvulsants for treatment and prevention of seizures. However it is frequently associated with drug-induced leukopenia. Hypersensitivity reactions to phenytoin are well recognized and can be severe. Phenytoin is associated with serious hematologic side effects such as agranulocytosis, thrombocytopenia, red cell aplasia and hemolytic anemia, either through humoral or cell-mediated immunemechanism. We describe a 57-year-old male patient who developed a severe granulocytopenia while taking phenytoin for 66 days in the total amount of 21.6 gram. Bone marrow examination showed isolated depletion of myeloid elements. After 10 days of phenytoin withdrawal and G-CSF treatment, the patient recovered from granulocytic suppression. Using in vitro culture, marrow suppression associated with phenytoin therapy was felt to be non-immune mediated marrow suppression.
Agranulocytosis ; Anemia, Hemolytic ; Anticonvulsants ; Bone Marrow Examination ; Bone Marrow* ; Granulocyte Colony-Stimulating Factor ; Humans ; Hypersensitivity ; Leukopenia ; Male ; Middle Aged ; Phenytoin* ; Seizures ; Thrombocytopenia

BACKGROUND: Donor leukocyte infusion (DLI) is an effective therapy for patients who relapse with leukemia after allogeneic bone marrow transplantation (BMT). This is due to the fact that the immune reactivity of infused allogeneic lymphocytes on relapsed leukemia cells plays a major role in the control of leukemia. However, severe graft-versus-host disease (GVHD) and pancytopenia compromise the success of this treatment in a substantial number of patients. METHODS: To evaluate the effect of DLI, we surveyed 6 BMT centers regarding their use of DLI for relapsed leukemia after BMT. Detailed forms were used to gather data regarding the original BMT, relapse, response to DLI, complication and survival. Reports of 11 patients were consequently available for analysis. RESULTS: Five (83.3%) of 6 patients with chronic myeloid leukemia (CML) achieved complete remission (CR) [time-to-CR; 116 (27~180) days after DLI], and currently 4 are alive in CR (49~436 days). Five patients (83.3%) developed GVHD, and 2 developed pancytopenia which was related to DLI. In acute leukemia, all patients received salvage chemotherapy prior to DLI. Only 1 of 3 patients with acute lymphoblastic leukemia (ALL) who had early relapse achieved CR, but durable remission was not yet confirmed (62+ days). Both 2 patients with acute myeloid leukemia (AML) achieved CR, and their CR durations were 242+ and 326 days after DLI, respectively. CONCLUSION: This study demonstrates that DLI can exert considerable effects against myeloid forms of leukemia, especially in CML. Further investigations of separating GVHD from the graft- versus-leukemia effect and finding more effective anti-leukemia approaches on acute leukemiaare necessary to improve the current DLI limitations.
Bone Marrow Transplantation* ; Bone Marrow* ; Drug Therapy ; Graft vs Host Disease ; Humans ; Leukemia* ; Leukemia, Myelogenous, Chronic, BCR-ABL Positive ; Leukemia, Myeloid, Acute ; Leukocytes* ; Lymphocytes ; Pancytopenia ; Precursor Cell Lymphoblastic Leukemia-Lymphoma ; Recurrence ; Tissue Donors*