Hypocellular acute myeloid leukemia treated with bone marrow transplantation.

Hypocellular acute myeloid leukemia (AML) mainly occurs in elderly patients, and is extremely rare in childhood. There is still no established treatment for hypocellular AML. We report the case of an 11-year-old boy with hypocellular AML who was treated successfully with allogenic bone marrow transplantation (allo-BMT). He presented with fever, pallor and pancytopenia. Bone marrow aspiration and biopsy confirmed a diagnosis of hypocellular AML. Although low-dose cytarabine induced reduction of blasts, it did not lead to complete remission. He subsequently received myeloablative conditioning and allo-BMT. Graft-versus-host disease (GVHD) prophylaxis included short-course methotrexate and cyclosporine. Neutrophil engraftment (>5 × 108 /L) and platelet recovery (>10 × 1010 /L) were achieved on days 13 and 27, respectively. He developed acute GVHD of the skin (grade 2), which responded well to treatment with prednisolone. He has remained in complete remission for 5 years since allo-BMT. We consider allo-BMT to be feasible for children with hypocellular AML.

[Efficacy of chemotherapy combined with bortezomib for two cases of relapsed/refractory acute lymphoblastic leukemia].

Bortezomib (BZM), a proteasome inhibitor, was recently reported to be effective against acute lymphoblastic leukemia (ALL). We report two cases of relapsed/refractory ALL, who were treated with BZM (1.3 mg/m2/dose, 2 doses/week for 2 weeks) in combination with vincristine, doxorubicin, dexamethasone, and L-asparaginase (L-ASP). The first patient was a 16-year-old girl who developed a bone marrow relapse 8 months after the initial diagnosis during consolidation chemotherapy. She received BZM-combined chemotherapy without L-ASP considering her previous history of an allergic reaction to L-ASP. The BZM-combined regimen was discontinued due to interstitial pneumonia development on day 13, and the interstitial pneumonia was successfully treated with steroid pulse therapy. Although her elevated serum LDH transiently normalized on day 16, blasts in peripheral blood did not disappear, and she died of leukemia without achieving remission. The second patient was a 17-year-old girl who developed a third bone marrow relapse after cord blood transplantation. She was given the same BZM combined regimen. Although the BZM-combined regimen was discontinued due to acute pancreatitis development on day 12, complete remission without platelet recovery was confirmed on day 62. Our experience suggests not only the effectiveness of BZM-combined chemotherapy but also the importance of controlling its toxicities when administered as a salvage therapy for advanced ALL patients.

High-dose chemotherapy followed by autologous peripheral blood stem cell transplantation for recurrent primary mediastinal malignant germ cell tumor: a case report.

A 15-yr-old boy presented with an anterior mediastinal mass, multiple lung metastases and obstruction of the left brachiocephalic vein, the superior vena cava and the subclavian vein. Tumor biopsy by CT guidance confirmed a diagnosis of GCT. Five courses of BEP therapy were performed, and CT of the chest revealed reduction in the anterior mediastinal mass and disappearance of the multiple lung metastases. We performed the anterior mediastinal mass extraction followed by adjuvant chemotherapy consisting of ICE and TIP. However, the AFP levels became elevated soon after. Abnormal accumulation was observed in the right upper lung by DW-MRI. After the operation, two courses of TI chemotherapy and two courses of HDCT followed by auto-PBSCT were performed. He was complicated with auditory disorder and renal dysfunction. Although HDCT followed by auto-PBSCT was effective for the relapsed primary mediastinal GCT, a treatment strategy avoiding late complications is warranted.

New series of antiprion compounds: pyrazolone derivatives have the potent activity of inhibiting protease-resistant prion protein accumulation.

To find effective antiprion compounds, we synthesized and evaluated various pyrazolone derivatives. Seven of 19 compounds showed inhibition of PrP-res accumulation and the remarkably active compound 13 showed an IC50 value of 3 nM in both ScN2a and F3 cell lines. Findings from studies on physicochemical and biochemical properties suggest that the action mechanism of these compounds does not correlate with any antioxidant activities, any of hydroxyl radical scavenging activities, or any SOD-like activities.