Eltrombopag Added to Immunosuppression in Severe Aplastic Anemia.

Peffault de Latour R, Kulasekararaj A, Iacobelli S, Terwel SR, Cook R, Griffin M, Halkes CJM, Recher C, Barraco F, Forcade E, Vallejo JC, Drexler B, Mear JB, Smith AE, Angelucci E, Raymakers RAP, de Groot MR, Daguindau E, Nur E, Barcellini W, Russell NH, Terriou L, Iori AP, La Rocca U, Sureda A, Sánchez-Ortega I, Xicoy B, Jarque I, Cavenagh J, Sicre de Fontbrune F, Marotta S, Munir T, Tjon JML, Tavitian S, Praire A, Clement L, Rabian F, Marano L, Hill A, Palmisani E, Muus P, Cacace F, Frieri C, van Lint MT, Passweg JR, Marsh JCW, Socié G, Mufti GJ, Dufour C, Risitano AM; Severe Aplastic Anemia Working Party of the European Society for Blood and Marrow Transplantation.N Engl J Med. 2022 Jan 6;386(1):11-23. doi: 10.1056/NEJMoa2109965.

Management of thrombosis in paroxysmal nocturnal hemoglobinuria: a clinician’s guide.

Griffin M, Munir T.

Ther Adv Hematol. 2017 Mar;8(3):119-126. doi: 10.1177/2040620716681748. Epub 2016 Dec 21.

SARS-CoV-2 infection in aplastic anemia.

Avenoso D, Marsh JCW, Potter V, Pagliuca A, Slade S, Dignan F, Tholouli E, Mittal S, Davis B, Tauro S, Kesse-Adu R, Griffin M, Payne E, Gandhi S, Kulasekararaj AG.

Haematologica. 2022 Feb 1;107(2):541-543. doi: 10.3324/haematol.2021.279928.

The incidence and prevalence of patients with paroxysmal nocturnal haemoglobinuria and aplastic anaemia PNH syndrome: A retrospective analysis of the UK’s population-based haematological malignancy research network 2004-2018.

Richards SJ, Painter D, Dickinson AJ, Griffin M, Munir T, Arnold L, Payne D, Pike A, Muus P, Hill A, Newton DJ, McKinley C, Jones R, Kelly R, Smith A, Roman E, Hillmen P.

Eur J Haematol. 2021 Aug;107(2):211-218. doi: 10.1111/ejh.13640. Epub 2021 Jun 9.

Long-term safety and efficacy of ravulizumab in patients with paroxysmal nocturnal hemoglobinuria: 2-year results from two pivotal phase 3 studies.

Kulasekararaj AG, Griffin M, Langemeijer S, Usuki K, Kulagin A, Ogawa M, Yu J, Mujeebuddin A, Nishimura JI, Lee JW, Peffault de Latour R; 301/302 Study Group.

Eur J Haematol. 2022 May 3. doi: 10.1111/ejh.13783.

Clinical and prognostic significance of small paroxysmal nocturnal hemoglobinuria clones in myelodysplastic syndrome and aplastic anemia.

Fattizzo B, Ireland R, Dunlop A, Yallop D, Kassam S, Large J, Gandhi S, Muus P, Manogaran C, Sanchez K, Consonni D, Barcellini W, Mufti GJ, Marsh JCW, Kulasekararaj AG.

Leukemia. 2021 Nov;35(11):3223-3231. doi: 10.1038/s41375-021-01190-9. Epub 2021 Mar 4.

Danicopan: an oral complement factor D inhibitor for paroxysmal nocturnal hemoglobinuria.

Risitano AM, Kulasekararaj AG, Lee JW, Maciejewski JP, Notaro R, Brodsky R, Huang M, Geffner M, Browett P.

Haematologica. 2021 Dec 1;106(12):3188-3197. doi: 10.3324/haematol.2020.261826.

Phase 2 study of danicopan in patients with paroxysmal nocturnal hemoglobinuria with an inadequate response to eculizumab.

Kulasekararaj AG, Risitano AM, Maciejewski JP, Notaro R, Browett P, Lee JW, Huang M, Geffner M, Brodsky RA.

Blood. 2021 Nov 18;138(20):1928-1938. doi: 10.1182/blood.2021011388.

Pegcetacoplan versus Eculizumab in PNH.

Kulasekararaj AG, Gandhi S, Brodsky RA.

N Engl J Med. 2021 Oct 28;385(18):1724-1725. doi: 10.1056/NEJMc2106424.

One-year outcomes from a phase 3 randomized trial of ravulizumab in adults with paroxysmal nocturnal hemoglobinuria who received prior eculizumab.

Kulasekararaj AG, Hill A, Langemeijer S, Wells R, González Fernández FA, Gaya A, Ojeda Gutierrez E, Piatek CI, Mitchell L, Usuki K, Bosi A, Brodsky RA, Ogawa M, Yu J, Ortiz S, Röth A, Lee JW, Peffault de Latour R.

Eur J Haematol. 2021 Mar;106(3):389-397. doi: 10.1111/ejh.13564. Epub 2021 Jan 3.

Monitoring of patients with paroxysmal nocturnal hemoglobinuria on a complement inhibitor.

Kulasekararaj AG, Brodsky RA, Hill A.

Am J Hematol. 2021 Jul 1;96(7):E232-E235. doi: 10.1002/ajh.26176. Epub 2021 Apr 14.

Categorizing hematological response to eculizumab in paroxysmal nocturnal hemoglobinuria: a multicenter real-life study.

Debureaux PE, Kulasekararaj AG, Cacace F, Silva BGP, Calado RT, Barone F, Sicre de Fontbrune F, Prata PH, Soret J, Sica M, Notaro R, Scheinberg P, Mallikarjuna V, Gandhi S, Large J, Risitano AM, Peffault de Latour R, Frieri C; Severe Aplastic Anemia Working Party of the European Group for Bone Marrow Transplantation.

Bone Marrow Transplant. 2021 Oct;56(10):2600-2602. doi: 10.1038/s41409-021-01372-0. Epub 2021 Jul 5.

PMID: 34226670

The importance of terminal complement inhibition in paroxysmal nocturnal hemoglobinuria.

Kulasekararaj AG, Brodsky RA, Nishimura JI, Patriquin CJ, Schrezenmeier H.

Ther Adv Hematol. 2022 May 30;13:20406207221091046. doi: 10.1177/20406207221091046. eCollection 2022.

Composite endpoint to evaluate complement inhibition therapy in patients with paroxysmal nocturnal hemoglobinuria.

Kulasekararaj A, Glasmacher A, Liu P, Szer J, Araten D, Rauch G, Gwaltney C, Sierra JR, Lee JW.

Eur J Haematol. 2022 May;108(5):391-402. doi: 10.1111/ejh.13746. Epub 2022 Mar 1.

Pegcetacoplan versus Eculizumab in PNH. Reply.

Hillmen P, Risitano AM, Peffault de Latour R.Hillmen P, et al. N Engl J Med. 2021 Oct 28;385(18):1725-1726. doi: 10.1056/NEJMc2106424.N Engl J Med. 2021. PMID: 34706185 No abstract available.

Effect of Eculizumab Treatment in Patients with Paroxysmal Nocturnal Hemoglobinuria with or without High Disease Activity: Real-World Findings from the International Paroxysmal Nocturnal Hemoglobinuria Registry.

Höchsmann B, Sicre de Fontbrune F, Lee JW, Kulagin AD, Hillmen P, Wilson A, Marantz JL, Schrezenmeier H.Höchsmann B, et al. Among authors: hillmen p. Eur J Haematol. 2022 Apr 7. doi: 10.1111/ejh.13773. Online ahead of print.Eur J Haematol. 2022. PMID: 35390189