Management of classical Philadelphia chromosome-negative myeloproliferative neoplasms in Asia: consensus of the Asian Myeloid Working Group.

Myeloproliferative neoplasms (MPN) are a heterogeneous group of clonal hematopoietic stem cell disorders characterized clinically by the proliferation of one or more hematopoietic lineage(s). The classical Philadelphia-chromosome (Ph)-negative MPNs include polycythemia vera (PV), essential thrombocythemia (ET) and primary myelofibrosis (PMF). The Asian Myeloid Working Group (AMWG) comprises representatives from fifteen Asian centers experienced in the management of MPN. This consensus from the AMWG aims to review the current evidence in the risk stratification and treatment of Ph-negative MPN, to identify management gaps for future improvement, and to offer pragmatic approaches for treatment commensurate with different levels of resources, drug availabilities and reimbursement policies in its constituent regions. The management of MPN should be patient-specific and based on accurate diagnostic and prognostic tools. In patients with PV, ET and early/prefibrotic PMF, symptoms and risk stratification will guide the need for early cytoreduction. In younger patients requiring cytoreduction and in those experiencing resistance or intolerance to hydroxyurea, recombinant interferon-α preparations (pegylated interferon-α 2A or ropeginterferon-α 2b) should be considered. In myelofibrosis, continuous risk assessment and symptom burden assessment are essential in guiding treatment selection. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) in MF should always be based on accurate risk stratification for disease-risk and post-HSCT outcome. Management of classical Ph-negative MPN entails accurate diagnosis, cytogenetic and molecular evaluation, risk stratification, and treatment strategies that are outcome-oriented (curative, disease modification, improvement of quality-of-life).

Clinical and laboratory profile of patients with thrombotic thrombocytopenic purpura seen at the University of Sto.Tomas Hospital: A single center experience

BACKGROUND: Thrombotic thrombocytopenic purpura (TTP) is considered a rare disease. In the Philippines, there are currently no local registry for such rare disease thus clinical features that may be unique to the country is difficult to characterize.OBJECTIVE: To characterize the TTP patients seen in our institution based on the demographic, clinical and laboratory profile of these patients.RESULTS: A total of eight patients were described in this report. Median age was 38 years (range, 20-63) with a female predominance. All patients had neurologic  symptoms and microangiopathic hemolytic anemia. Minor neurologic symptoms (confusion, headache and dizziness) were more frequently observed. Renal dysfunction (serum creatinine >1.4mg/dL), fever (>38.0°C), and thrombocytopenia were observed in four patients (50%), seven patients (87.5%) and six patients (75%) respectively. Patients underwent median of three sessions of Plasma Exchange (range, two to 38). Of the six patients who received therapeutic plasma exchange, five were discharged alive.CONCLUSION: Fifty percent of the patients in this report presented with the classic pentad. This feature should be kept in mind as TTP is a true hematopologic emergency. A high index of suspicion, prompt evaluation and treatment of patients are necessary because multi-organ thrombosis may not be reversible. If treatment is not urgently administered, Therapeutic Plasma Exchange is the management of choice and should be done immediately once TTP is recognized.

Clinical profile and survival of Filipino myelofibrosis patients seen in a tertiary hospital

BACKGROUND: Myelofibrosis (MF) is a disease characterized by panmyelosis with intact maturation, progressive bone marrow fibrosis, and splenomegaly with extramedullary hematopoiesis. It is the most heterogeneous disease in the group of myeloproliferative neoplasms in terms of patient characteristics and natural history of the illness. Rationale andOBJECTIVES: This study aims to describe the clinical, laboratory profile and survival of myelofibrosis patients seen in our institution and also to descriptively compare these characteristics of Filipino MF patients to Chinese and White MF patients.RESEARCH DESIGN AND METHODOLOGY: An analysis of patients' data retrospectively collected through review of clinical records from 1994 to 2012 was done. All patients diagnosed with primary myelofibrosis (PMF) or post-polycythemia (post-PV) myelofibrosis or post-essential thrombocytosis (post-ET) myelofibrosis were included in this study. Survival was measured from first day of diagnosis to death from any cause or last known follow-up or direct communication to patient/relatives. Survival data were estimated using the Kaplan-Meier method. We compared the clinical characteristics and laboratory profile at diagnosis of these Filipino myelofibrosis patients to the Chinese as reported by Xu et. al., and White MF patients as reported by Cervantes et al.RESULTS/SUMMARY: In our patients' data set, they seem to be younger with a median age of 57, less likely having constitutional symptoms but they have higher frequency of anemia, splenomegaly and leukocytosis. The heterogeneity of myelofibrosis' clinical and laboratory features were seen in this epidemiologic study on Filipino myelofibrosis patients in comparison with Chinese and White MF patients. The clinical course is a slowly progressive chronic disease with a median survival probability of 180 months.

Successful treatment of thrombotic thrombocytopenic purpura with a month-long therapeutic plasma exchange and immunosuppressive agents

OBJECTIVES: To describe the clinical course and treatment response of a case of thrombotic thrombocytopenic purpura (TTP) after a month of therapeutic plasma exchange (TPE) and immunosuppressive agents and to review related published literature regarding TTP and its response to TPE and immunosuppressive agents.CASE AND DISCUSSION: A 64-year-old female presented with fever, bicytopenia, change in sensorium, and seizure of one day duration. Metabolic panel showed normal electrolytes with normal brain imaging. Hematologic work-up showed anemia, thrombocytopenia and leukocytosis with normal differential count. Reticulocyte was elevated. Peripheral smear showed significant schistocytes with nucleated red blood cells and marked thrombocytopenia. Thrombotic thrombocytopenic purpura was the initial consideration with the fulfilment of four out of the pentad of TTP, namely: microangiopathichaemolytic anemia, thrombocytopenia, fever and neurologic symptoms. Therapeutic plasma exchange was immediately initiated with daily platelet count and Lactate dehydrogenase (LDH) determination to assess response. However, despite daily plasma exchanges and continuous plasma infusion, there was inadequate response. In this light, immunosuppressive agents were started in the following order: high dose methylprednisolone, weekly rituximab and intravenous cyclophosphamide. On the 31st hospital day, after daily TPE and combined immunosuppression, she achieved complete response with a platelet count of 170 X 109/L to 250 X 109/L (baseline-14 X 109/L) and LDH 227U/L (baseline-1,191 U/L).CONCLUSION: This study presented a challenging case of TTP which was successfully treated with the standard of care together with the available adjunctive treatment options.