Pericardial effusion (PcE) is one of the uncommon complications after hematopoietic stem cell transplantation (HSCT). Although many causes are related with PcE after HSCT, PcE after HSCT is usually late-onset and can be presented as a sign of acute or chronic graft-versus-host disease in allogeneic transplantation. Previous reports of PcE after autologous HSCT are very uncommon. Transplantation-associated thrombotic microangiopathy (TA-TMA) is a kind of renal microvascular complications after HSCT, which is similar to thrombotic thrombocytopenic purpura. The authors report a case of early-onset PcE, which maybe resulted from TA-TMA, after high-dose chemotherapy and autologous peripheral blood HSCT in a 4-year-old child with neuroblastoma.
Child ; Drug Therapy ; Graft vs Host Disease ; Hematopoietic Stem Cell Transplantation ; Hematopoietic Stem Cells ; Humans ; Neuroblastoma ; Pericardial Effusion ; Preschool Child ; Purpura, Thrombotic Thrombocytopenic ; Thrombotic Microangiopathies ; Transplantation, Homologous

Objective: To observe the efficacy and adverse reactions of a combination therapy regimen based on bortezomib and glucocorticoids in recurrent/refractory immune thrombocytopenic purpura (iTTP) . Methods: Six patients with recurrent/refractory TTP were included and treated with a glucocorticoid and two courses of bortezomib-based regimen. The clinical remission status of patients, changes in ADAMTS13 activity/ADAMTS13 inhibitor, and the occurrence of treatment-related adverse reactions were observed. Results: Of the 6 patients, 2 were males and 4 were females, with a median age of 21.5 (18-68) years. Refractory TTP was found in 1 case and recurrent TTP in 5 cases. Glucocorticoids were administered with reference to prednisone at 1 mg·kg(-1)·d(-1), and gradually reduced in dosage after achieving clinical remission. Bortezomib is subcutaneously administered at 1.3 mg/m(2) on days 1, 4, 8, and 11 with a 28-day treatment course consisting of 2 courses. Six patients achieved clinical remission after receiving bortezomib as the main treatment. ADMATS13 activity returned to normal in all patients with TTP after treatment, and the ADAMTS13 inhibitor turned negative. Thrombocytopenia is the most common adverse reaction after treatment, with other adverse reactions, including peripheral neuritis and abdominal pain, but ultimately all patients returned to normal. In a median follow-up of 26 (9-41) months, 5 patients maintained sustained remission, and 1 patient relapsed after 16 months of bortezomib treatment. Conclusion: Combination therapy of bortezomib and glucocorticoids has a satisfactory therapeutic effect and controllable adverse reactions for recurrent/refractory iTTP.
Male ; Female ; Humans ; Young Adult ; Adult ; Middle Aged ; Aged ; Bortezomib/therapeutic use* ; Glucocorticoids/therapeutic use* ; Rituximab/therapeutic use* ; Purpura, Thrombotic Thrombocytopenic/drug therapy* ; Purpura, Thrombocytopenic, Idiopathic/drug therapy* ; ADAMTS13 Protein/therapeutic use*

As extra-mammary Paget's disease is rare and usually diagnosed at early stage when it is highly curable with surgical resection, it is much rarer to see patients with recurrent metastatic disease. Thrombotic thrombocytopenic purpura in patients with metastatic solid cancer is also a rare disease and may result from bone marrow metastasis or bone marrow necrosis. For the latter, the majority of cases are not eligible for systemic chemotherapy for rapid disease progression and poor performance status. Herein, authors report a patient with thrombotic thrombocytopenic purpura associated with bone marrow necrosis complicating extra-mammary Paget's disease who was successfully treated with docetaxel and carboplatin combination chemotherapy.
Bone Marrow* ; Carboplatin ; Disease Progression ; Drug Therapy ; Drug Therapy, Combination ; Humans ; Necrosis* ; Neoplasm Metastasis ; Paget Disease, Extramammary* ; Purpura, Thrombotic Thrombocytopenic* ; Rare Diseases

OBJECTIVETo evaluate the efficacy and safety of rituximab in treating patients with refractory and/or relapsing thrombotic thrombocytopenic purpura (TTP).

METHODSTotally three patients received rituximab as salvage therapy in our hospital. Rituximab was administered at a weekly dose of 375 mg/m(2) for 2 or 4 consecutive weeks. After clinical remission, patients were followed up every 3 months.

RESULTSAll three patients achieved complete remission. The median time to platelet count recovery was 7 days (4-12 days) after the first rituximab infusion. During the follow-up (median: 12 months; range: 9-18 months), no patients experienced relapse. No side effect was noted during treatment and follow-up period.

CONCLUSIONTherapy with rituximab is effective and well tolerated for patients with refractory or relapsing TTP.

Adult ; Aged ; Antibodies, Monoclonal, Murine-Derived ; therapeutic use ; Female ; Humans ; Middle Aged ; Purpura, Thrombotic Thrombocytopenic ; drug therapy ; Retrospective Studies ; Rituximab ; Salvage Therapy ; Treatment Outcome

The outcomes of the treatment of thrombotic thrombocytopenic purpura (TTP) have been shown to be improved by the administration of plasma exchange. However, treatment options are currently limited for cases refractory to plasma exchange. The autoantibodies that block the activity of ADAMTS13 have been demonstrated to play a role in the pathogenesis of TTP; therefore, high-dose immunoglobulin, which can neutralize these autoantibodies, may be useful for refractory TTP. However, successful treatment with high-dose immunoglobulin for TTP refractory to plasma exchange and corticosteroids has yet to be reported in Korea. Herein, we describe a refractory case which was treated successfully with high-dose immunoglobulin. A 29-year-old male diagnosed with TTP failed to improve after plasma exchange coupled with additional high-dose corticosteroid therapy. As a salvage treatment, we initiated a 7-day regimen of high-dose immunoglobulin (400 mg/kg) infusions, which resulted in a complete remission, lasting up to the last follow-up at 18 months. High-dose immunoglobulin may prove to be a useful treatment for patients refractory to plasma exchange; it may also facilitate recovery and reduce the need for plasma exchange.
Adrenal Cortex Hormones/*therapeutic use ; Adult ; Humans ; Immunoglobulins/administration & dosage/*therapeutic use ; Male ; *Plasma Exchange ; Purpura, Thrombotic Thrombocytopenic/*drug therapy ; Recurrence/prevention & control ; Salvage Therapy ; Treatment Failure

Wegener's granulomatosis is a distinct form of necrotizing granulomatous vasculitis which usually affects the kidneys and the upper and lower respiratory tracts. Unusual manifestations have also been reported, and these include colitis, urethritis and diabetes insipidus. We describe a case of Wegener's granulomatosis which presented with rapidly progressive renal insufficiency, sudden deafness, red eye, facial palsy, and complicated by uncommon manifestations that were diffuse pulmonary hemorrhage and thrombotic thrombocytopenic purpura.
Aged ; Cyclosporine/therapeutic use ; Female ; Hemorrhage/complications* ; Human ; Lung Diseases/complications* ; Prednisolone/therapeutic use ; Purpura, Thrombotic Thrombocytopenic/complications* ; Wegener's Granulomatosis/drug therapy ; Wegener's Granulomatosis/diagnosis ; Wegener's Granulomatosis/complications*

Androgen Antagonists ; therapeutic use ; Anilides ; therapeutic use ; Antineoplastic Agents, Hormonal ; therapeutic use ; Bone Neoplasms ; complications ; secondary ; Goserelin ; therapeutic use ; Humans ; Male ; Middle Aged ; Nitriles ; therapeutic use ; Prostatic Neoplasms ; complications ; drug therapy ; Purpura, Thrombotic Thrombocytopenic ; drug therapy ; etiology ; Tosyl Compounds ; therapeutic use