Successful peripheral blood stem cell transplantation for myelodysplastic syndrome

Wilms' tumor (WT1) gene expression is increased in patients with leukemia as well as myelodysplastic syndrome (MDS) and is useful for detection of minimal residual disease (MRD). A 47-year-old man given a diagnosis of refractory anemia with excess of blasts in transformation (RAEB-T) received myeloablative therapy followed by autologous peripheral blood stem cell transplantation (PBSCT). MRD by WT1 expression was not detected in the graft. The patient has been in CR for 25 months after PBSCT. These observations suggest that PBSCT is feasible for patients with RAEB-T and analysis of WT1 expression can be applied for patients with high risk MDS.     

Pericardial graft vs. host disease in a patient with myelodysplastic syndrome following peripheral blood stem cell transplantation

A patient with myelodysplastic syndrome developed pericardial effusion 20 month after allogenic peripheral blood stem cell transplantation. Sclerotic and erythematous skin lesions were observed over the face and extremities, and a diagnosis of chronic graft vs. host disease (GVHD) was made based on skin biopsy findings. Pericardial fluid contained numerous CD8+/HLA-DR+ lymphocytes, but no leukaemic cells. Tumour necrosis factor alpha (TNFalpha) and soluble Fas (sFas) levels were highly elevated in both the effusion and serum. The patient was treated with methylprednisolone and tacrolimus. Skin GVHD improved rapidly associated with resolution of pericardial effusion and reductions in cytokine levels. We concluded that pericardial effusion was due to pericarditis and was a manifestation of chronic GVHD in this patient, and that cytotoxic lymphocytes and specific cytokines played significant roles.     

Early onset invasive pulmonary zygomycosis following allogeneic peripheral blood stem cell transplantation in a patient with therapy-related myelodysplastic syndrome

A 39-year-old woman received an allogeneic peripheral blood stem cell transplantation from her daughter for secondary myelodysplastic syndrome. On day +12, a cough and fever developed. Chest X-ray and computed tomography demonstrated a consolidation in the left lung. A diagnosis of pulmonary zygomycosis was made on the histology from a transbronchial lung biopsy. Although amphotericin B (AMPH-B) showed efficacy, dose reduction was necessary because of renal toxicity. The patient died of pulmonary zygomycosis, confirmed by an autopsy. We strongly hope for authorization as soon as possible of the use of such a drug as liposomal AMPH-B which appears less toxic.     

Dissimilar course in the right and left ventricular function in a patient with dilated cardiomyopathy

The case of a patient with severe dilative cardiomyopathy and cardiomegaly demonstrates that M-mode echocardiography and planar chest radiographs are not always sufficient to follow up the disease correctly. Using other non-invasive imaging modalities, in this case radionuclide ventriculography and NMR tomography, which provide more precise information with regard to morphology and function of both the right and the left heart, allows a more reliable follow-up of the disease.     

Successful reduced-intensity stem cell transplantation in a patient with myelodysplastic syndrome combined with Sweet's syndrome

A 53-year-old male with myelodysplastic syndrome developed Sweet's syndrome extensively over his left iliac and inguinal regions that was refractory to standard treatment with corticosteroids and chemotherapy, received a stem cell transplant from an HLA-matched unrelated donor, conditioned by reduced-intensity regimen. The patient achieved complete hematological remission, and the cutaneous lesions improved gradually and then disappeared completely despite the patient receiving granulocyte colony-stimulating factor after transplantation and developing acute graft-versus-host disease.     

Transient left ventricular aneurysm in a patient with hypertrophic cardiomyopathy and myocarditis

Left ventricular wall motion abnormalities, aneurysm formation, and progression to global hypokinesis have been described in patients with myocarditis and in patients with hypertrophic cardiomyopathy. We document a case of reversible aneurysm formation, cardiogenic shock, and complete recovery in a patient with myocarditis and hypertrophic cardiomyopathy. Pathophysiologic mechanisms of myocardial injury and recovery are discussed.     

Marked improvement of left ventricular function after parathyroidectomy in a hemodialysis patient with secondary hyperparathyroidism and left ventricular dysfunction.

A 52-year-old woman, a hemodialysis patient, was admitted because of exertional dyspnea. Echocardiography showed left ventricular (LV) dilatation and reduced contraction. Coronary angiography showed no fixed stenosis. She had elevated levels of parathyroid hormone (PTH) as a result of secondary hyperparathyroidism with advanced renal failure. After parathyroidectomy, marked improvement of LV function following immediate decrease of blood levels of PTH was observed. It is suggested that PTH might have a significant role in the pathogenesis of LV dysfunction and that parathyroidectomy might be effective as a therapy for heart failure in some patients with secondary hyperparathyroidism and LV dysfunction.     

Second allogeneic peripheral blood stem cell transplantation with fludarabine-based low-intensity conditioning regimen for relapsed myelodysplastic syndrome after allogeneic bone marrow transplantation

We describe the case of a 51-year-old patient with relapsed myelodysplastic syndrome after allogeneic bone marrow transplantation (BMT), who underwent allogeneic peripheral blood stem cell transplantation (PBSCT) after conditioning with a novel regimen consisting of fludarabine, busulfan, and antithymocyte globulin. The second PBSCT was performed early, at 3 months after the initial allogeneic BMT, but it was well tolerated and complete hematologic remission was documented. The patient did not experience any early transplantation-related organ toxicity but died from opportunistic infection 6 months after the second transplantation. Our experience suggests that this novel regimen may induce remission and could be offered to patients relapsing after the first transplantation; however, the fludarabine-containing regimen might be accompanied by profound immunosuppression.     

Allogeneic peripheral blood stem cell transplantation in an elderly patient with myelodysplatic syndrome with myelofibrosis

Allogeneic peripheral blood stem cell transplantation (Allo-PBSCT) has in recent years become an alternative to allogeneic bone marrow transplantation because it facilitates rapid hematopoietic reconstitution without an increase in the incidence of severe graft-versus-host disease (GVHD). We report on a 61-year-old man with myelodysplastic syndrome (MDS) and myelofibrosis who received an allo-PBSCT from his HLA-matched 68-year-old brother. The preparative regimen consisted of busulfan and cyclophosphamide. Cyclosporin A and methotrexate were administered for GVHD prophylaxis. The donor was treated with granulocyte colony-stimulating factor (G-CSF) at a dose of 10 micrograms/kg/day subcutaneously for 4 consecutive days. A preparation of 4.04 x 10(6) CD34+ cells/kg recipient weight was collected in a single apheresis and infused immediately. Engraftment times to a neutrophil count greater than 500/microliter and platelet count greater than 2.0 x 10(4)/microliter were 15 days each. Acute GVHD of grade II developed, but was resolved with methylprednisolone. However, the patient died of thrombotic microangiopathy 97 days after his allo-PBSCT. Administration of G-CSF and apheresis in the donor were feasible and well tolerated. Allo-PBSCT may result in earlier engraftment and be especially beneficial to elderly patients with MDS.     

多普勒超声检测心肌运动指数评价扩张型心肌病左心功能

目的 :探讨超声检测心肌运动指数对评价扩张型心肌病 (DCM )左心功能的临床价值。方法 :DCM患者 31例 ,正常对照者 6 2例 ,应用多普勒超声心动图记录二尖瓣舒张期和左心室流出道收缩期脉冲多普勒血流频谱 ,测量心肌运动指数。结果 :①与正常对照组相比 ,DCM组等容舒张期时间 [(98.39± 2 0 .83)ms∶(6 6 .4 5±11.32 )ms ,P <0 .0 1]及等容收缩期时间 [(39.5 2± 13.31)ms∶(2 1.4 5± 7.6 5 )ms,P <0 .0 1]明显延长、射血时间[(2 2 3.39± 4 0 .93)ms∶(2 73.0 6± 2 1.0 1)ms ,P <0 .0 1]明显缩短 ,导致心肌运动指数 (0 .6 3± 0 .14∶0 .32± 0 .0 4 ,P <0 .0 1)明显升高 ,相关分析表明 ,心肌运动指数与左心功能障碍程度呈正相关 ;②相关分析显示 ,心肌运动指数与年龄、心率、血压无相关性。结论 :①心肌运动指数是评价DCM患者左心功能简便而准确的多普勒超声新指标 ,且与左心功能障碍程度呈正相关 ;②心肌运动指数不受年龄、心率、血压的影响     

Recurrence of monoclonal gammopathy associated with donor-derived myelodysplastic syndrome after cord blood stem cell transplantation

Myelodysplastic syndrome (MDS) is known to be associated with functional abnormalities of B cells, including hypergammaglobulinemia and monoclonal gammopathy (MG). However, the pathogenesis of these immunological disorders has not been clarified. We report a patient who developed donor-derived MDS followed by leukemic transformation after cord blood transplantation for MDS with MG. Interestingly, MG reappeared before development of donor-derived MDS. We analyzed the immunoglobulin allotype gene polymorphisms to determine whether the MG after cord blood transplantation was of recipient origin or donor origin. Results of genetic analysis and enzyme-linked immunosorbent assay of IgG1 allotype revealed that the MG after cord blood transplantation was of donor origin. Although the mechanism of donor-derived MG remains unclear, the persistent presence of recipient's antigen presenting cells might have induced the abnormal immunoglobulin production.