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Objective: Allogeneic hematopoietic stem cell transplantation (allo-SCT) is a potentially curative treatment for severe aplastic anemia (SAA). This is a single institutional review to study the feasibility of using allo-SCT for Thai children with SAA., Material and Method: Nine children with SAA (7 matched-sibling donor-SCT, 1 matched-unrelated donor-SCT and 1 haploidentical-SCT) underwent allo-SCT between October 2002 and September 2010. Cyclophosphamide and anti-thymocyte globulin (CY/ATG) were used as conditioning regimen for 4 patients with matched-sibling donor-SCT CY/ATG and fludarabine were used for 3 patients with matched-sibling donor-SCT and one patient with haplo-identical SCT. One matched-unrelated donor-SCT received CY/ATG and total body irradiation., Results: Eight of 9 patients (89%) achieved neutrophil engraftment within 13.5 days (range 6.0-22.0). One matched-sibling donor-SCT recipient who failed to achieve engraftment died from acute renal failure and gram-negative sepsis on day 21 post allo-SCT. One matched-sibling donor-SCT case developed late graft failure on day 72 and died from invasive fungal infection. For graft versus host disease (GVHD), a haplo-identical-SCT patient died from steroid refractory grade IV acute GVHD. At last follow-up, six patients (67%) alive at a median follow-up time of 76.4 months (range 2.3-88.8). Overall survival (OS) and event-free survival (EFS) at 5 year was 63% and 65%, respectively., Conclusion: Allo-SCT is a feasible curative treatment for children with SAA in Thailand. Graft failure and severe GVHD in alternative donors SCT are responsible for major causes of death. OS and EFS probabilities are stable after the first year post transplant.

Background: Congenital or acquired prothrombin deficiency is a rare condition., Case Report: A 2-year-7-month old Thai girl presented with ecchymosis, bleeding at both thighs and right ear lobe after a self-limited viral infection., Results: The investigations revealed prolonged APTT and PT, prothrombin level 6% and positive anticardiolipin antibody 26.2 IU/mL. The 1:1 mixture of her plasma and normal plasma could not normalize her APTT and PT. The inhibitor to prothrombin determined by Bethesda method was 0.62 BU. She was responsive to 20 ml/kg of FFP transfusion, followed by 10 ml/kg at an interval of 12 h for three days and daily 500 units of prothrombin complex concentrate administration for three days. At two-week follow-up, she had no bleeding symptom, coagulation tests were normal, prothrombin level was normalized at 94%, no inhibitor to prothrombin was detected, and anticardiolipin antibody became negative. The additional DNA analysis of her prothrombin gene revealed nine different polymorphisms for which seven had been found in patients with congenital prothrombin deficiency and two were novel (4096T-->C, 4097T-->C). These single nucleotide polymorphisms are not the disease-causing mutations. In addition, neither known mutations inducing congenital prothrombin deficiency were identified., Conclusion: The acquired hypoprothrombinemia was concluded as the cause of bleeding in this reported patient. It might be caused by the transient low titer of antiphospholipid antibody, which was responsive to replacement therapy of FFP and prothrombin complex concentrate.

Background: Alveolar rhabdomyosarcoma is a primitive malignant round cell neoplasm, which shows skeletal muscle differentiation. Although their histopathologic and immunohistochemical findings are well known, the cytology, immunocytochemistry and molecular study on pleural effusion have not been well documented., Objective: To apply molecular method in the diagnosis and monitoring of alveolar rhabdomyosarcoma., Case Report: The case of a 14-year-old Thai male, who presented with dyspnea and left pleural effusion. Computed tomography of the chest and abdomen showed a huge heterogeneous enhancing mass at the left retroperitoneum. Pleural fluid cytology showed malignant small round blue cells. Immunocytochemical stains on cell block material showed positive reactivity to vimentin, sarcomeric actin, desmin, MyoD1, myogenin, and CD56 in round cell tumor Reverse transcription-polymerase chain reaction (RT-PCR) demonstrated PAX/FKHR fusion transcript. The patient received chemotherapeutic regimen for advanced-stage rhabdomyosarcoma. Finally, he succumbed to the disease, thirteen months after the diagnosis., Conclusion: Immunocytochemistry on cell block in conjunction with determination of PAX/FKHR fusion mRNA by RT-PCR is a molecular method in the diagnosis and monitoring of alveolar rhabdomyosarcoma inpleural fluid.