Prolonged response after stopping TPO-RA in ITP, TP53 mutations and myelofibrosis outcomes, clinical picture of ERCC6L2 disease, where variants predispose to marrow failure and malignancy

In this week’s episode we’ll discuss the findings from a prospective trial assessing response after discontinuation of TPO-receptor agonist therapy in primary ITP, learn more about the impact of TP53 on the outcomes of patients with myelofibrosis following hematopoietic stem cell transplant, and review the comprehensive clinical picture of the ERCC-6L2 disease, where germline variants predispose to marrow failure and myeloid malignancies.

Released June 8, 2023

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The articles highlighted in this episode are listed below:

• Prolonged response after TPO-RA discontinuation in primary ITP: results of a prospective multicenter study

  Authors: Stéphanie Guillet, Etienne Crickx, Imane Azzaoui, Pascal Chappert, Emmanuelle Boutin, Jean-François Viallard, Etienne Rivière, Delphine Gobert, Lionel Galicier, Marion Malphettes, Stéphane Cheze, François Lefrere, Sylvain Audia, Bernard Bonnotte, Olivier Lambotte, Nicolas Noel, Olivier Fain, Guillaume Moulis, Mohamed Hamidou, Mathieu Gerfaud-Valentin, Jean-Pierre Marolleau, Louis Terriou, Nihal Martis, Anne-Sophie Morin, Antoinette Perlat, Thomas Le Gallou, Frédérique Roy-Peaud, Ailsa Robbins, Jean-Christophe Lega, Matthieu Puyade, Thibault Comont, Nicolas Limal, Laetitia Languille, Anissa Zarrour, Marine Luka, Mickael Menager, Thibault Belmondo, Sophie Hue, Florence Canoui-Poitrine, Marc Michel, Bertrand Godeau, Matthieu Mahévas

• TPO-RAs and ITP remission: cause or coincidence?

  Authors: Kristen M. Sanfilippo, Adam Cuker

• Impact of TP53 on outcome of patients with myelofibrosis undergoing hematopoietic stem cell transplantation

  Authors: Nico Gagelmann, Anita Badbaran, Rachel B. Salit, Thomas Schroeder, Carmelo Gurnari, Simona Pagliuca, Victoria Panagiota, Christina Rautenberg, Bruno Cassinat, Felicitas Thol, Christine Wolschke, Marie Robin, Michael Heuser, Marie-Thérèse Rubio, Jaroslaw P. Maciejewski, Hans Christian Reinhardt, Bart L. Scott, Nicolaus Kröger

• Double, double, TP53, and trouble in myelofibrosis

  Author: Pramila Krishnamurthy

• The clinical picture of ERCC6L2 disease: from bone marrow failure to acute leukemia

  Authors: Marja Hakkarainen, Ilse Kaaja, Suvi P. M. Douglas, Tom Vulliamy, Inderjeet Dokal, Jean Soulier, Lise Larcher, Régis Peffault de Latour, Thierry Leblanc, Flore Sicre de Fontbrune, Timo Siitonen, Olli Lohi, Eva Hellström-Lindberg, Gisela Barbany, Bianca Tesi, Akiko Shimamura, Fabian Beier, Sharon Jackson, Amir Asher Kuperman, Tzipora Falik Zaccai, Hannah Tamary, Cristina Mecucci, Ilaria Capolsini, Kirsi Jahnukainen, Urpu Salmenniemi, Riitta Niinimäki, Teppo Varilo, Outi Kilpivaara, Ulla Wartiovaara-Kautto

• ERCC6L2 syndrome: attack of the TP53 clones

  Author: Marcin W. Wlodarski