Objectives: The complement component 5 (C5) inhibitor ravulizumab demonstrated non-inferiority to eculizumab following 26 weeks of treatment in complement inhibitor-naïve and complement inhibitor-experienced patients with paroxysmal nocturnal hemoglobinuria (PNH; studies 301 and 302, respectively). This study aims to describe the results of both studies from 27 weeks to 2 years.Methods: Patients (N = 441) continued to receive ravulizumab throughout the extension period. Efficacy endpoints included lactate dehydrogenase (LDH) normalization, transfusion avoidance and fatigue score (FACIT-F). Safety analyses were also performed.Results: From 27 weeks to 2 years, improvements in LDH levels were maintained in both study populations. Transfusion avoidance was maintained in 81.9% (study 301) and 85.6% (study 302) of patients, and FACIT-F scores remained stable. Ravulizumab was well tolerated, and the incidence of adverse events (AEs) were similar between patients of both studies. Incidence of serious AEs deemed related to ravulizumab treatment was low (<3%).Conclusions: This study reports, to date, the longest period of follow-up in over 400 patients with PNH treated with ravulizumab (662 patient-years). Long-term, ravulizumab demonstrated durable efficacy and was well tolerated, highlighting the importance of C5 inhibitors as the mainstay of PNH treatment.

BackgroundThe evolution of molecular studies in myeloproliferative neoplasms (MPN) has enlightened us the understanding of this complex disease consisting of polycythaemia vera (PV), essential thrombocythemia (ET) and primary myelofibrosis (PMF). The epidemiology is well described in the western world but not in Asian countries like Malaysia.Materials and methodsThis retrospective national registry of MPN was conducted from year 2009 to 2015 in Malaysia.ResultsA total of 1010 patients were registered over a period of 5 years. The mean age was 54 years with male predominance. The ethnic distribution revealed that Chinese had a relatively high weighted incidence proportion (43.2%), followed by Indian (23.8%), Malay (15.8%) and other ethnic groups (17.2%). The types of MPN reported were 40.4% of ET (n = 408), 38.1% of PV (n = 385), 9.2% of PMF (n = 93), 3.1% of hypereosinophilic syndrome (HES) (n = 31) and 7.9% of unclassifiable MPN (MPN-U) (n = 80). Splenomegaly was only palpable clinically in 32.2% of patients. The positive JAK2 V617F mutation was present in 644 patients with 46.6% in PV, 36.0% in ET, 9.0% in PMF, and 7.4% in MPN-U, and had significantly lower haemoglobin (p < 0.001), haematocrit (p < 0.001) and white blood cells (WBC) (p < 0.001) than those with negative mutation. Significant differences in platelet and WBC count were detected in ethnic groups and MPN sub-types. There were more arterial thrombosis events seen in those with JAK2 V617F mutation as compared to venous thrombosis events (23.1% vs 4.4%). The bleeding rate was only 6.6%. Among the risk factors, previous thrombosis, old age (≥ 60 years) and hypertension were significantly correlated to positive JAK2 V617F mutation. The arterial thrombosis event is associated with higher presenting HB, HCT and PLT while the bleeding event is associated with lower presenting HB, HCT but higher PLT. The presence of JAK2 V617F mutation is associated with higher risk of arterial thrombosis.ConclusionChinese ethnicity is associated with higher rates of MPN. The history of thrombosis, age ≥ 60 years and hypertension are risk factors that can be correlated to JAK2 V617F mutation. This study is instrumental for policy makers to ensure preventive strategies can be implemented in future.

BackgroundClassical Hodgkin lymphoma (cHL) is a clinicopathologically unique, aggressive lymphoma arising from germinal center B-cells and is one of the most curable hematological malignancies. This study aimed to determine the clinical course, treatment regimens, response rates, and survival data of patients diagnosed with cHL in a tertiary center.MethodsA retrospective review was conducted to include patients with a diagnosis of cHL from 2013 to 2017. Data of demographic and clinical characteristics, treatment regimens, and outcomes were collected and analyzed.ResultsWe recruited 94 patients with a median age of 27.0 [interquartile range (IQR), 12] years. Most of the patients were male (61.7%) and 73.4% were ethnic Malay. Nodular sclerosis was the most common histology (77.6%), followed by mixed cellularity (6.4%) and others (16%). The median follow-up time was 28.0 (IQR, 32) months. All patients received chemotherapy but only 13.8% received radiotherapy as consolidation. The doxorubicin-bleomycin-vinblastine-dacarbazine regimen was the most common (85.1%), followed by the escalated bleomycin-etoposide-doxorubicin-cyclophosphamide-vincristineprednisolone-procarbazine regimen (14.9%). Following treatment, 76.1% of patients achieved complete response. The 2-year overall survival (OS) and progression-free survival (PFS) of the entire cohort were 96.5% and 71.1%, respectively. The 2-year OS and PFS for advanced-stage disease were 93.9% and 62.8%, compared to 100% and 82.7% for early-stage disease, respectively (P=0.252 and P=0.052, respectively).ConclusionThis study provides insight into the clinical presentation and treatment outcomes among patients with cHL in Malaysia. A longer study duration is required to identify OS and PFS benefits and treatment-related complications for different chemotherapeutic regimens.

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