Consensus recommendations for optimising the diagnosis and treatment of paroxysmal nocturnal haemoglobinuria in Singapore.

Introduction: Paroxysmal nocturnal haemoglobinuria (PNH) is a rare haematologic disease characterised by intravascular haemolysis, thrombophilia and bone marrow failure. There is a lack of established clinical guidance on the screening, diagnosis and manage-ment of PNH in Singapore. A relatively low level of awareness among healthcare professionals regarding PNH manifestations further contributes to diagnostic delays. Additionally, limited access to complement inhibitors, like eculizumab, may delay treatment and impact patient outcomes. Method: Nine haematologists from different institu-tions in Singapore convened to formulate evidence-based consensus recommendations for optimising the diagnosis and management of patients with PNH and improving access to novel treatments. The experts reviewed the existing literature and international guidelines published from January 2010 to July 2023, focusing on 7 clinical questions spanning PNH screening, diagnostic criteria, investigations, treatment and monitoring of subclinical and classic disease, PNH with underlying bone marrow disorders, and PNH in pregnancy. A total of 181 papers were reviewed to formulate the statements. All experts voted on the statements via 2 rounds of Delphi and convened for an expert panel discussion to finetune the recommendations. Results: Sixteen statements have been formulated for optimising the screening, diagnosis and management of PNH. Upon confirmation of PNH diagnosis, individuals with active haemolysis and/or thrombosis should be considered for anti-complement therapy, with eculizumab being the only approved drug in Singapore. Conclusion: The current recommendations aim to guide the clinicians in optimising the screening, diagnosis and management of PNH in Singapore.

Long-term follow-up of Asian patients younger than 46 years with acute myeloid leukemia in first complete remission: comparison of allogeneic vs. autologous hematopoietic stem cell transplantation.

Optimal therapy for patients with acute myeloid leukemia (AML) in first complete remission (CR-1) remains the subject of debate: with the possibilities of chemotherapy alone, autologous (auto) or allogeneic (allo) hematopoietic stem cell transplantation (HSCT). We undertook a retrospective analysis of AML patients aged below 46 years and in CR-1, who had undergone auto- or allo-HSCT in our institution, to determine the factors associated with a better outcome. Between September 1985 and April 2003, 81 patients underwent autologous (n = 29) or allogeneic (n = 52) HSCT in CR-1. With a median follow-up of 10 years, the probability of 15-year overall survival (OS) was 51%[95% confidence interval (CI) = 32 - 70%] for auto-HSCT vs. 55% (95% CI = 42 - 69) for allo-HSCT, respectively (P = 0.92). The cumulative incidence of relapse at 3 years was 49% (95% CI = 30 - 67) (auto) vs. 21% (95% CI = 10 - 30) (allo), respectively. Non-relapse-related mortality at 3 years was 5% (95% CI = 0 - 14) (auto) vs. 17% (95% CI = 7 - 28) (allo), respectively. This retrospective analysis confirmed that auto- and allo-HSCT led to similar OS. The significantly lower incidence of relapse amongst allograft recipients was balanced by an increased incidence of death in CR.