Efficacy of rabbit antithymocyte globulin as first-line treatment of severe aplastic anemia: an Asian multicenter retrospective study

Due to the unavailability of horse antithymocyte globulin (ATG) in many markets worldwide, patients with severe aplastic anemia (SAA) are limited to the use of rabbit ATG. We aimed to analyze hematologic response and overall survival (OS) of Asian patients treated with rabbit ATG as first-line therapy of SAA. We retrospectively reviewed the medical records of 97 consecutive patients who received rabbit ATG as first-line treatment of SAA from 2006 to 2012 at centers in four Asian countries. The primary endpoint was 6- and 12-month overall response rates (ORR) for patients receiving rabbit ATG within the recommended dose range (2.5-3.75 mg/kg/day). Secondary endpoints included ORR in patients receiving any dose of rabbit ATG and 2-year OS. For patients who received rabbit ATG within the recommended dose range, 6- and 12-month ORRs were 17.4 and 63.6 %, respectively. For patients who received any dose of rabbit ATG, 6- and 12-month ORRs were 24.3 and 68.6 %, respectively. The 2-year OS rate was 86.3 %. Rabbit ATG is effective for treatment of SAA in Asian patients. The 12-month ORR and 2-year OS with rabbit ATG were comparable to historical results obtained with horse ATG

A Retrospective Multicenter Study to Evaluate the Efficacy of Rabbit Antithymocyte Globulin (rATG) Immunosuppressive Therapy As First-Line Treatment of Aplastic Anemia

TesisEl objetivo principal de las acciones proyectadas en la presente investigación aplicada en Gerencia Pública, es crear valor público a través de la gestión empresarial de la EPS SEDAPAR Arequipa, con la reducción de pérdidas de agua potable que contribuya a mejorar y hacer sostenible la calidad de vida de la población de Arequipa. Obedece a la necesidad de lograr la eficiencia en la administración del agua potable producida, desde los puntos de vista: ambiental, técnico y comercial, obteniendo como resultado de ello la reducción del indicador de agua no facturada mediante la identificación y la propuesta de soluciones a los eventos generadores de pérdidas de agua. Entre las estrategias propuestas se destacan la sectorización hidráulica de la red, el control de las presiones y el control de las conexiones con uso indebido

Review of disease-related complications and management in adult patients with thalassemia: A multi-center study in Thailand.

Disease-related complications and management are different among patients with thalassemia. This study was aimed to review the prevalence, clinical risk factors for the complications and the management in patients with thalassemia in Thailand. A multicenter cross-sectional study was conducted in patients with thalassemia aged ≥ 18 years old. Thalassemia-related complications and management were reviewed. The clinical parameters significantly associated with the complications were analyzed by logistic regression methods. The prevalence of thalassemia-related complications was 100% in patients with transfusion-dependent thalassemia (TDT) and 58.8% in patients with non-transfusion-dependent thalassemia (NTDT). Advanced age was statistically associated with extramedullary hematopoiesis in both TDT and NTDT patients. Splenectomy was a significant risk factor for pulmonary hypertension in both groups of patients. Severe iron overload started earlier in patients with TDT than NTDT and was associated with diabetes mellitus (adjusted odds ratio (AOR) = 6.2, p-value = 0.02). Disease-related complications are more prevalent in patients with TDT than patients with NTDT. Splenectomy and advanced age were important risk factors for developing major complications in both groups. Early screening and management for specific disease-related complications should be considered in patients with thalassemia according to their clinical risk factors

Secondary dengue serotype 1 infection causing dengue shock syndrome with rhombencephalitis and bleeding associated with refractory thrombocytopenia: A case report

Background: Dengue has a wide spectrum of manifestations, from an asymptomatic condition to dengue shock syndrome. Extensive plasma leakage, severe bleeding, or both, could lead to dengue shock syndrome, a common cause of death in dengue-infected patients. Thrombocytopenia is a common laboratory finding in dengue, which correlates with the disease severity and rapidly resolves during the recovery phase. Therefore, refractory thrombocytopenia is rare in patients with dengue. Rhombencephalitis is an inflammatory disease affecting the hindbrain, rarely associated with dengue. We report the second case of dengue-associated rhombencephalitis, wherein the patient developed dengue shock syndrome and severe bleeding associated with refractory thrombocytopenia. Case report: A 47-year-old Thai female with secondary dengue serotype 1 infection developed dengue shock syndrome with rhombencephalitis, manifested as altered sensorium and status epilepticus in the critical phase. Cerebrospinal fluid analysis showed pleocytosis with predominantly mononuclear cells and high protein levels. Magnetic resonance imaging of the brain showed multifocal brain signal abnormalities involving the medulla oblongata, pons, midbrain, bilateral hippocampi, thalami, posterior limb of internal capsules, external capsules, and deep hemispheric white matter. The patient had partial neurological recovery following rhombencephalitis for one month. During the recovery phase, severe bleeding with refractory thrombocytopenia and acute kidney injury were observed. Methylprednisolone with eltrombopag was administered, which resulted in an increased the platelet count, cessation of bleeding and recovery of kidney function within 4 days. Conclusions: Dengue is a potential cause of rhombencephalitis. Dengue-associated rhombencephalitis develops during the critical phase, with only partial neurological recovery. However, severe bleeding and refractory thrombocytopenia were also observed during the recovery phase. Methylprednisolone with a thrombopoietin receptor agonist could be an effective treatment for increasing platelet count and stopping bleeding in dengue

Defining serum ferritin thresholds to predict clinically relevant liver iron concentrations for guiding deferasirox therapy when MRI is unavailable in patients with non-transfusion-dependent thalassaemia

Liver iron concentration (LIC) assessment by magnetic resonance imaging (MRI) remains the gold standard to diagnose iron overload and guide iron chelation therapy in patients with non-transfusion-dependent thalassaemia (NTDT). However, limited access to MRI technology and expertise worldwide makes it practical to also use serum ferritin assessments. The THALASSA (assessment of Exjade((R)) in non-transfusion-dependent THALASSemiA patients) study assessed the efficacy and safety of deferasirox in iron-overloaded NTDT patients and provided a large data set to allow exploration of the relationship between LIC and serum ferritin. Using data from screened patients and those treated with deferasirox for up to 2years, we identified clinically relevant serum ferritin thresholds (for when MRI is unavailable) for the initiation of chelation therapy (>800g/l), as well as thresholds to guide chelator dose interruption (2000g/l). (clinicaltrials.gov identifier: NCT00873041)

Deferasirox demonstrates a dose‐dependent reduction in liver iron concentration and consistent efficacy across subgroups of non‐transfusion‐dependent thalassemia patients

The 1-year THALASSA study enrolled 166 patients with various non-transfusion-dependent thalassemia (NTDT) syndromes, degrees of iron burden and patient characteristics, and demonstrated the overall efficacy and safety of deferasirox in reducing liver iron concentration (LIC) in these patients. Here, reduction in LIC with deferasirox 5 and 10 mg/kg/day starting dose groups is shown to be consistent across the following patient subgroups—baseline LIC/serum ferritin, age, gender, race, splenectomy (yes/no), and underlying NTDT syndrome (β-thalassemia intermedia, HbE/β-thalassemia or α-thalassemia). These analyses also evaluated deferasirox dosing strategies for patients with NTDT. Greater reductions in LIC were achieved in patients dose-escalated at Week 24 from deferasirox 10 mg/kg/day starting dose to 20 mg/kg/day. Patients who received an average actual dose of deferasirox >12.5–≤17.5 mg/kg/day achieved a greater LIC decrease compared with the ≥7.5–≤12.5 mg/kg/day and >0–<7.5 mg/kg/day subgroups, demonstrating a dose–response efficacy. LIC reduction across patient subgroups was generally consistent with the primary efficacy analysis with a similar safety profile. Am. J. Hematol. 88:503–506, 2013. © 2013 Wiley Periodicals, Inc