Predictors for improvement in patient-reported outcomes: post hoc analysis of a phase 3 randomized, open-label study of eculizumab and ravulizumab in complement inhibitor-naive patients with paroxysmal nocturnal hemoglobinuria. Schrezenmeier H, Kulasekararaj A, Mitchell L, de Latour RP, Devos T, Okamoto S, Wells R, Popoff E, Cheung A, Wang A, Tomazos I, Patel Y, Lee JW. Ann Hematol. 2023 Oct 7. doi: 10.1007/s00277-023-05483-0. Online ahead of print. PMID: 37804344

Risk factors for thromboembolic events in patients with paroxysmal nocturnal hemoglobinuria (PNH): a nested case-control study in the International PNH Registry. Höchsmann B, Peffault de Latour R, Hill A, Röth A, Devos T, Patriquin CJ, Chou WC, Jain D, Zu K, Wu C, Lee JW.Ann Hematol. 2023 Nov;102(11):2979-2988. doi: 10.1007/s00277-023-05402-3. Epub 2023 Sep 5. PMID: 37668788

SARS-CoV-2 vaccination in 361 non-transplanted patients with aplastic anemia and/or paroxysmal nocturnal hemoglobinuria. Griffin M, Eikema DJ, Verheggen I, Kulagin A, Tjon JM, Fattizzo B, Ingram W, Zaidi U, Desnica L, Giammarco S, Drozd-Sokolowska J, Xicoy B, Patriarca A, Loschi M, Szmigielska-Kaplon A, Beier F, Cignetti A, Drexler B, Gavriilaki E, Lanza F, Orvain C, Risitano AM, De la Camara R, De Latour RP. Haematologica. 2023 Aug 17. doi: 10.3324/haematol.2023.283863. Online ahead of print. PMID: 37584297

Graft- versus-host disease and relapse/rejection-free survival after allogeneic transplantation for idiopathic severe aplastic anemia: a comprehensive analysis from the SAAWP of the EBMT. Devillier R, Eikema DJ, Dufour C, Aljurf M, Wu D, Maschan A, Kulagin A, Halkes CJM, Collin M, Snowden J, Renard C, Ganser A, Sykora KW, Gibson BE, Maertens J, Itäla-Remes M, Corti P, Cornelissen J, Bornhäuser M, Araujo MC, Ozdogu H, Risitano A, Socie G, De Latour RP. Haematologica. 2023 Sep 1;108(9):2305-2315. doi: 10.3324/haematol.2022.281876.PMID: 36951165

A clickable melphalan for monitoring DNA interstrand crosslink accumulation and detecting ICL repair defects in Fanconi anemia patient cells. Berrada S, Martínez-Balsalobre E, Larcher L, Azzoni V, Vasquez N, Da Costa M, Abel S, Audoly G, Lee L, Montersino C, Castellano R, Combes S, Gelot C, Ceccaldi R, Guervilly JH, Soulier J, Lachaud C. Nucleic Acids Res. 2023 Aug 25;51(15):7988-8004. doi: 10.1093/nar/gkad559. PMID: 37395445

Biochemical phenotyping of paroxysmal nocturnal hemoglobinuria reveals solute carriers and β-oxidation deficiencies. Yamakawa PE, Fonseca AR, Guerreiro da Silva IDC, Gonçalves MV, Marchioni DM, Carioca AAF, Michonneau D, Arrais-Rodrigues C. PLoS One. 2023 Aug 1;18(8):e0289285. doi: 10.1371/journal.pone.0289285. eCollection 2023. PMID: 37527257

Clinical and Molecular Determinants of Clonal Evolution in Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria. Gurnari C, Pagliuca S, Prata PH, Galimard JE, Catto LFB, Larcher L, Sebert M, Allain V, Patel BJ, Durmaz A, Pinto AL, Inacio MCB, Hernandez L, Dhedin N, Caillat-Zucman S, Clappier E, Sicre de Fontbrune F, Voso MT, Visconte V, Peffault de Latour R, Soulier J, Calado RT, Socié G, Maciejewski JP. J Clin Oncol. 2023.PMID: 36054881

Complement inhibition in medicine: Hematology and beyond; complement inhibition in hematology: PNH and beyond. Peffault de Latour R, Risitano AM.Am J Hematol. 2023. PMID: 36917199

Haemolytic paroxysmal nocturnal haemoglobinuria in patients with myeloid neoplasms: A rare association with specific therapeutic implications. Sutra Del Galy A, Willems L, D’Aveni M, Pautas C, Chantepie S, Carpentier B, Barraco F, Banos A, Garidi R, Forcade E, Sicre de Fontbrune F, Peffault de Latour R.Br J Haematol. 2023.PMID: 36807116

Rare germline complement factor H variants in patients with paroxysmal nocturnal hemoglobinuria; Prata PH, Galimard JE, Sicre de Fontbrune F, Duval A, Vieira Martins P, Roncelin S, Debureaux PÉ, Lepretre AC, Larcher L, Birsen R, Benhamou Y, Soulier J, Socié G, Fremeaux-Bacchi V, Peffault de Latour R.Blood. 2023.PMID: 36626252

Clonal hematopoiesis driven by chromosome 1q/MDM4 trisomy defines a canonical route toward leukemia in Fanconi anemia; Sebert M, Gachet S, Leblanc T, Rousseau A, Bluteau O, Kim R, Ben Abdelali R, Sicre de Fontbrune F, Maillard L, Fedronie C, Murigneux V, Bellenger L, Naouar N, Quentin S, Hernandez L, Vasquez N, Da Costa M, Prata PH, Larcher L, et al. J.Cell Stem Cell. 2023. PMID: 36736290

Phase 3 Study of Subcutaneous Versus Intravenous Ravulizumab in Eculizumab-Experienced Adult Patients with PNH: Primary Analysis and 1-Year Follow-Up. Yenerel MN, Sicre de Fontbrune F, Piatek C, Sahin F, Füreder W, Ortiz S, Ogawa M, Ozol-Godfrey A, Sierra JR, Szer J.Adv Ther. 2023 PMID: 36272026

The Clinical Picture of the ERCC6L2 Disease – from Bone Marrow Failure to Acute Leukemia. Hakkarainen M, Kaaja I, Douglas SPM, Vulliamy TJ, Dokal I, Soulier J, Larcher L, Peffault de Latour R, Leblanc TM, Sicre de Fontbrune F, Siitonen T, Lohi O, Hellström-Lindberg E, Barbany G, Tesi B, Shimamura A, Beier F, Jackson SR, Kuperman A, Falik Zaccai TC, Tamary H, Mecucci C, Capolsini I, Jahnukainen K, Salmenniemi U, Niinimäki R, Varilo T, Kilpivaara O, Wartiovaara-Kautto U. Blood. 2023. PMID: 36952636

A novel cancer risk prediction score for the natural course of FA patients with biallelic BRCA2/FANCD1 mutations. Radulovic I, Schündeln MM, Müller L, Ptok J, Honisch E, Niederacher D, Wiek C, Scheckenbach K, Leblanc T, Larcher L, Soulier J, Reinhardt D, Schaal H, Andreassen PR, Hanenberg H.Hum Mol Genet. 2023. PMID: 36721989

Current use of androgens in bone marrow failure disorders: a report from the Severe Aplastic Anemia Working Party of the European Society of Blood and Marrow Transplantation. Pagliuca S, Kulasekararaj AG, Eikema DJ, Piepenbroek B, Iftikhar R, Satti TM, Griffin M, Laurino M, Kupesiz A, Bertrand Y, Fattizzo B, Yakoub-Agha I, Aljurf M, Corti P, Massaccesi E, Lioure B, Calabuig M, Klammer M, Unal E, Wu D, Chevallier P, Forcade E, Snowden JA, Ozdogu H, Risitano A, De Latour RP. Haematologica. 2023.PMID: 37199126 

Molecular landscape of immune pressure and escape in aplastic anemia. Pagliuca S, Gurnari C, Hercus C, Hergalant S, Nadarajah N, Wahida A, Terkawi L, Mori M, Zhou W, Visconte V, Spellman S, Gadalla SM, Zhu C, Zhu P, Haferlach T, Maciejewski JP. Leukemia. 2023.PMID: 36253429

Mitigating Drug-Target-Drug Complexes in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Switch C5 Inhibitors. Nishimura JI, Soubret A, Arase N, Buatois S, Hotta M, Charoin JE, Ito Y, Sreckovic S, Takamori H, Bucher C, Ueda Y, Hernández-Sánchez J, Gotanda K, Jordan G, Shinomiya K, Ramos J, Kim JS, Panse J, de Latour RP, Röth A, Morii E, Schrezenmeier H, Isaka Y, Sica S, Kanakura Y, Yoon SS, Kinoshita T, Paz-Priel I, Sostelly A.Clin Pharmacol Ther. 2023 .PMID: 36660902

Graft-versus-host disease and relapse/rejection-free survival after allogeneic transplantation for idiopathic severe aplastic anemia: a comprehensive analysis from the SAAWP of the EBMT. Devillier R, Eikema DJ, Dufour C, Aljurf M, Wu D, Maschan A, Kulagin A, Halkes CJM, Collin M, Snowden J, Renard C, Ganser A, Sykora KW, Gibson BE, Maertens J, Itäla-Remes M, Corti P, Cornelissen J, Bornhäuser M, Araujo MC, Ozdogu H, Risitano A, Socie G, De Latour RP.Haematologica. 2023 .PMID: 36951165

Concomitant Immunosuppressive Therapy and Eculizumab Use in Patients with Paroxysmal Nocturnal Hemoglobinuria: An International PNH Registry Analysis. Hill A, de Latour RP, Kulasekararaj AG, Griffin M, Brodsky RA, Maciejewski JP, Marantz JL, Gustovic P, Schrezenmeier H.Acta Haematol. 2023. PMID: 36108594 

IPSS-M in myelodysplastic neoplasms arising from aplastic anemia and paroxysmal nocturnal hemoglobinuria. Gurnari C, Prata PH, Catto LFB, Durmaz A, Larcher L, Sébert M, Allain V, Kewan T, Pagliuca S, Pinto AL, B Inacio MC, Hernandez L, Dhedin N, Caillat-Zucman S, Clappier E, Sicre de Fontbrune F, Voso MT,Visconte V, Peffault de Latour R, Soulier J, Socie G, Calado RT, Maciejewski JP .Blood. 2023. PMID: 37053552