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001-es BibID:	BIBFORM115904
035-os BibID:	(cikkazonosító)e963 (WoS)001096083700001 (Scopus)85175712230
Első szerző:	Kiladjian, Jean-Jacques
Cím:	Momelotinib in Myelofibrosis Patients With Thrombocytopenia : Post Hoc Analysis From Three Randomized Phase 3 Trials / Kiladjian Jean-Jacques, Vannucchi Alessandro M., Gerds Aaron T., Gupta Vikas, Verstovsek Srdan, Egyed Miklos, Platzbecker Uwe, Mayer Jirí, Grosicki Sebastian, Illés Árpád, Woźny Tomasz, Oh Stephen T., McLornan Donal, Kirgner Ilya, Yoon Sung-Soo, Harrison Claire N., Klencke Barbara, Huang Mei, Kawashima Jun, Mesa Ruben
Dátum:	2023
ISSN:	2572-9241
Megjegyzések:	The oral activin A receptor type I, Janus kinase 1 (JAK1), and JAK2 inhibitor momelotinib demonstrated symptom, spleen, and anemia benefits in intermediate- and high-risk myelofibrosis (MF). Post hoc analyses herein evaluated the efficacy and safety of momelotinib in patients with MF and thrombocytopenia (platelet counts <100 x 109/L) from randomized phase 3 studies: MOMENTUM (momelotinib versus danazol; JAK inhibitor experienced); SIMPLIFY-1 (momelotinib versus ruxolitinib; JAK inhibitor naive); and SIMPLIFY-2 (momelotinib versus best available therapy; JAK inhibitor experienced); these studies were not statistically powered to assess differences in thrombocytopenic subgroups, and these analyses are descriptive. The treatment effect of momelotinib versus ruxolitinib on week 24 response rates (spleen volume reduction >= 35%/Total Symptom Score reduction >= 50%/transfusion independence) was numerically comparable or better in thrombocytopenic patients versus the overall JAK inhibitor naive population; rates were preserved with momelotinib in thrombocytopenic patients but attenuated with ruxolitinib (momelotinib: 27%/28%/67% overall versus 39%/35%/61% in thrombocytopenic group; ruxolitinib: 29%/42%/49% overall versus 0%/22%/39% in thrombocytopenic group, respectively). In contrast to ruxolitinib, momelotinib maintained high dose intensity throughout the treatment. In the JAK inhibitor experienced population, thrombocytopenic patients had the following: (1) numerically higher symptom and transfusion independence response rates with momelotinib than in control arms; and (2) preserved spleen, symptom, and transfusion independence response rates with momelotinib relative to the overall study populations. The safety profile of momelotinib in thrombocytopenic patients was also consistent with the overall study population. In summary, momelotinib represents a safe and effective treatment option for patients with MF and moderate-to-severe thrombocytopenia.
Tárgyszavak:	Orvostudományok Klinikai orvostudományok idegen nyelvű folyóiratközlemény külföldi lapban folyóiratcikk MOMELOTINIB MYELOFIBROSIS
Megjelenés:	HemaSphere. - 7 : 11 (2023), p. 1-15. -
További szerzők:	Vannucchi, Alessandro Maria Gerds, Aaron T. Gupta, Vikas Verstovsek, Srdan Egyed Miklós Platzbecker, Uwe Mayer, Jiri Grosicki, Sebastian Illés Árpád (1959-) (belgyógyász, haematológus, onkológus) Woźny, Tomasz Oh, Stephen T. McLornan, Donal P. Kirgner, Ilya Yoon, Sung-Soo Harrison, Claire N. Klencke, Barbara J. Huang, Mei Kawashima, Jun Mesa, Ruben
Internet cím:	Szerző által megadott URL DOI Intézményi repozitóriumban (DEA) tárolt változat
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001-es BibID:	BIBFORM114431
035-os BibID:	(WoS)000921253000001 (Scopus)85146688051
Első szerző:	Waszczuk-Gajda, Anna
Cím:	Safety and efficacy of autologous stem cell transplantation in dialysis-dependent myeloma patients - The DIADEM study from the chronic malignancies working party of the EBMT / Anna Waszczuk-Gajda, Luuk Gras, Liesbeth C. de Wreede, Tiarlan Sirait, Arpad Illes, Zubeyde Nur Ozkurt, John A. Snowden, Mutlu Arat, Claude Eric Bulabois, Judith Niederland, Matjaz Sever, Shankara Paneesha, Victoria Potter, Alain Gadisseur, Thomas Chalopin, Gwendolyn Van Gorkom, Joaquin Martínez López, Tessa Kerre, Joanna Drozd-Sokolowska, Kavita Raj, Patrick J. Hayden, Meral Beksac, Ibrahim Yakoub-Agha, Donal P. McLornan, Stefan Schönland
Dátum:	2023
ISSN:	0268-3369
Megjegyzések:	The role of high-dose chemotherapy followed by autologous stem cell transplantation (ASCT) in the treatment of myeloma (MM) patients with severe and/or dialysis-dependent renal impairment remains uncertain. We report on the outcomes of 110 patients (median age 57 years) who had become dialysis-dependent pre-ASCT and who underwent a first ASCT between 1997 and 2017. Sixty-three (57%) patients had light chain MM. All patients required dialysis (94% hemodialysis and 6% peritoneal). Forty-four of 71 (62%) patients received bortezomib-based induction regimens and 42 (39%) patients had achieved at least a very good partial response (VGPR) pre-ASCT. Melphalan dosing was as follows: <= 140 mg/m(2) (82%), and >140 mg/m(2) (18%). The median PFS after ASCT was 35 months (95% CI: 21.5-42.2) and the median OS 102 months (95% CI: 70.4-129.1). At 1, 2, and 5 years after ASCT, 8% (95% CI 3-14%), 13% (6-20%), and 20% (12-29%) of patients, respectively, had achieved dialysis independence. In multivariate analyses of OS and PFS including age at ASCT, response at ASCT, and year of ASCT, younger age at ASCT and better response at ASCT (CR/VGPR/PR vs. MR/SD/progression) were significantly associated with better OS and PFS.
Tárgyszavak:	Orvostudományok Elméleti orvostudományok idegen nyelvű folyóiratközlemény külföldi lapban folyóiratcikk
Megjelenés:	Bone Marrow Transplantation. - 58 : 4 (2023), p. 424-429. -
További szerzők:	Gras, Luuk Wreede, Liesbeth C. de Sirait, Tiarlan Illés Árpád (1959-) (belgyógyász, haematológus, onkológus) Ozkurt, Zubeyde Nur Snowden, John A. Arat, Mutlu Bulabois, Claude Eric Niederland, Judith Sever, Matjaz Paneesha, Shankara Potter, Victoria Gadisseur, Alain Chalopin, Thomas Gorkom, Gwendolyn Van López, Joaquin Martínez Kerre, Tessa Drozd-Sokolowska, Joanna Raj, Kavita Hayden, Patrick J. Beksac, Meral Yakoub-Agha, Ibrahim McLornan, Donal P. Schönland, Stefan
Internet cím:	Szerző által megadott URL DOI Intézményi repozitóriumban (DEA) tárolt változat
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