Online first
Wytyczne / stanowisko ekspertów
Opublikowany online: 2024-12-30
Wyświetlenia strony
83
Wyświetlenia/pobrania artykułu
154
Priorytety refundacyjne w hematologii – co udało się zrealizować w 2024 roku, a co jeszcze przed nami?
Streszczenie
Brak
Słowa kluczowe: wytycznestanowisko ekspertów
Referencje
- Montesinos P, Recher C, Vives S, et al. Ivosidenib and azacitidine in IDH1-mutated acute myeloid leukemia. N Engl J Med. 2022; 386(16): 1519–1531.
- Botton SDe, Montesinos P, Polo SV, et al. Updated efficacy and safety data from the AGILE study in patients with newly diagnosed acute myeloid leukemia treated with ivosidenib + azacitidine compared to placebo + azacitidine. J Clin Oncol. 2023; 41(16_suppl): 7012–7012.
- Erba HP, Montesinos P, Kim HJ, et al. QuANTUM-First Study Group. Quizartinib plus chemotherapy in newly diagnosed patients with FLT3-internal-tandem-duplication-positive acute myeloid leukaemia (QuANTUM-First): a randomised, double-blind, placebo-controlled, phase 3 trial. Lancet. 2023; 401(10388): 1571–1583.
- Platzbecker U, Della Porta MG, Santini V, et al. Efficacy and safety of luspatercept versus epoetin alfa in erythropoiesis-stimulating agent-naive, transfusion-dependent, lower-risk myelodysplastic syndromes (COMMANDS): interim analysis of a phase 3, open-label, randomised controlled trial. Lancet. 2023; 402(10399): 373–385.
- Rea D, Lang F, Kim DW, et al. Asciminib, a specific allosteric BCR-ABL1 inhibitor, in patients with chronic myeloid leukemia carrying the T315I mutation in a phase 1 trial. Blood. 2018; 132(Suppl 1): 792–792.
- Sayyed A, Kim TS, Park S et al. Somatic mutations in myeloid transcription factors and in activated signaling pathway, but not in epigenetic modifier pathway, predict the risk of treatment failure and progression to advanced phase in chronic myeloid leukemia. ASH2023, Abstract no: 4949.
- Gupta V, Harrison C, Gorsh B, et al. Red blood cell transfusion independence status is an independent predictor of survival: a post hoc time-dependent analysis of the phase 3 simplify-1, simplify-2, and momentum trials. Blood. 2023; 142(Supplement 1): 3188–3188.
- Vachhani P, Yacoub A, Traer E, et al. Platelet response in pacritinib-treated patients with cytopenic myelofibrosis: a retrospective analysis of PERSIST-2 and PAC203 studies. Blood. 2023; 142(Suppl. 1): 4554–4554.
- Elli EM, Di Veroli A, Bartoletti D, et al. Deferasirox in the management of iron overload in patients with myelofibrosis treated with ruxolitinib: the multicentre retrospective RUX-IOL study. Br J Haematol. 2022; 197(2): 190–200.
- Gill H, Au L, Tsai D, et al. S211: efficacy and safety of ropeginterferon alfa-2b for pre-fibrotic primary myelofibrosis and dipss low/intermediate-1 risk myelofibrosis: updated results and genomic characteristics. HemaSphere. 2023; 7(S3): e9247372.
- Beauverd Y, Ianotto JC, Thaw K, et al. Impact of cytoreductive drugs upon outcomes in a contemporary cohort of adolescent and young adults with essential thrombocythemia and polycythemia vera. Blood. 2023; 142(Suppl. 1): 748–748.
- Litzow M, Sun Z, Paietta E, et al. Consolidation therapy with blinatumomab improves overall survival in newly diagnosed adult patients with b-lineage acute lymphoblastic leukemia in measurable residual disease negative remission: results from the ECOG-ACRIN E1910 randomized phase III National Cooperative Clinical Trials Network trial. Blood. 2022; 140(Suppl 2): LBA-1–LBA-1.
- Shah BD, Ghobadi A, Oluwole OO, et al. KTE-X19 anti-CD19 CAR T-cell therapy in adult relapsed/refractory acute lymphoblastic leukemia: ZUMA-3 phase 1 results. Blood. 2021; 138(1): 11–22.
- Shadman M. Diagnosis and treatment of chronic lymphocytic leukemia: a review. JAMA. 2023; 329(11): 918–932.
- Hus I, Giannopoulos K, Jamroziak K, et al. Diagnostic and therapeutic recommendations of the Polish Society of Haematologists and Transfusiologists, and Polish Adult Leukemia Group-CLL for chronic lymphocytic leukemia in 2023. Acta Haematol Pol. 2023; 54(6): 342–371.
- Eichhorst B, Ghia P, Niemann CU, et al. ESMO Guidelines Committee. Electronic address: clinicalguidelines@esmo.org. ESMO Clinical Practice Guideline interim update on new targeted therapies in the first line and at relapse of chronic lymphocytic leukaemia. Ann Oncol. 2024; 35(9): 762–768.
- Wierda WG, Brown J, Abramson JS, et al. Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma, Version 2.2024, NCCN Clinical Practice Guidelines in Oncology. J Natl Compr Canc Netw. 2024; 22(3): 175–204.
- Kater AP, Owen C, Moreno C, et al. Fixed-duration ibrutinib-venetoclax in patients with chronic lymphocytic leukemia and comorbidities. NEJM Evid. 2022; 1(7): EVIDoa2200006.
- Tam CS, Allan JN, Siddiqi T, et al. Fixed-duration ibrutinib plus venetoclax for first-line treatment of CLL: primary analysis of the CAPTIVATE FD cohort. Blood. 2022; 139(22): 3278–3289.
- Budde LE, Sehn LH, Matasar M, et al. Safety and efficacy of mosunetuzumab, a bispecific antibody, in patients with relapsed or refractory follicular lymphoma: a single-arm, multicentre, phase 2 study. Lancet Oncol. 2022; 23(8): 1055–1065.
- Zinzani PL, Mayer J, Flowers CR, et al. ROSEWOOD: a phase II randomized study of zanubrutinib plus obinutuzumab versus obinutuzumab monotherapy in patients with relapsed or refractory follicular lymphoma. J Clin Oncol. 2023; 41(33): 5107–5117.
- Opat S, Tedeschi A, Linton K, et al. The MAGNOLIA trial: zanubrutinib, a next-generation bruton tyrosine kinase inhibitor, demonstrates safety and efficacy in relapsed/refractory marginal zone lymphoma. Clin Cancer Res. 2021; 27(23): 6323–6332.
- Opat S, Tedeschi A, Hu B, et al. Safety and efficacy of zanubrutinib in relapsed/refractory marginal zone lymphoma: final analysis of the MAGNOLIA study. Blood Adv. 2023; 7(22): 6801–6811.
- Neelapu SS, Chavez JC, Sehgal AR, et al. Long-term safety and activity of axicabtagene ciloleucel in refractory large B-cell lymphoma (ZUMA-1): a single-arm, multicentre, phase 1-2 trial. Lancet Oncol. 2019; 20(1): 31–42.
- Dreyling M, Fowler NH, Dickinson M, et al. Tisagenlecleucel in adult relapsed or refractory follicular lymphoma: the phase 2 ELARA trial. Nat Med. 2022; 28(2): 325–332.
- Neelapu SS, Chavez JC, Sehgal AR, et al. Three-year follow-up analysis of axicabtagene ciloleucel in relapsed/refractory indolent non-Hodgkin lymphoma (ZUMA-5). Blood. 2024; 143(6): 496–506.
- Neelapu S, Chavez J, Sehgal A, et al. Axicabtagene ciloleucel (axi-cel) in patients with relapsed/refractory indolent non-Hodgkin lymphoma: 4-year follow-up from the phase 2 ZUMA-5 trial. Blood. 2023; 142(Suppl. 1): 4868–4868.
- Dreyling M, Fowler NH, Dickinson M, et al. Durable response after tisagenlecleucel in adults with relapsed/refractory follicular lymphoma: ELARA trial update. Blood. 2024; 143(17): 1713–1725.
- Ansell SM, Radford J, Connors JM, et al. ECHELON-1 Study Group, ECHELON-1 Study Group. Brentuximab vedotin with chemotherapy for stage III or IV Hodgkin's lymphoma. N Engl J Med. 2018; 378(4): 331–344.
- Ansell SM, Radford J, Connors JM, et al. ECHELON-1 Study Group. Brentuximab vedotin with chemotherapy for stage III/IV classical Hodgkin lymphoma: 3-year update of the ECHELON-1 study. Blood. 2020; 135(10): 735–742.
- Borchmann P, Ferdinandus J, Schneider G, et al. German Hodgkin Study Group, Swiss Group for Clinical Cancer Research, Arbeitsgemeinschaft Medikamentöse Tumortherapie, Nordic Lymphoma Group, Australasian Leukaemia and Lymphoma Group. Assessing the efficacy and tolerability of PET-guided BrECADD versus eBEACOPP in advanced-stage, classical Hodgkin lymphoma (HD21): a randomised, multicentre, parallel, open-label, phase 3 trial. Lancet. 2024; 404(10450): 341–352.
- Rutherford S, Li H, Herrera A, et al. Nivolumab-AVD is better tolerated and improves progression-free survival compared to bv-avd in older patients (aged ≥60 years) with advanced stage hodgkin lymphoma enrolled on SWOG S1826. Blood. 2023; 142(Suppl 1): 181–181.
- Bartlett NL, Chen R, Fanale MA, et al. Retreatment with brentuximab vedotin in patients with CD30-positive hematologic malignancies. J Hematol Oncol. 2014; 7: 24.
- van Imhoff GW, McMillan A, Matasar MJ, et al. Ofatumumab versus rituximab salvage chemoimmunotherapy in relapsed or refractory diffuse large b-cell lymphoma: the ORCHARRD study. J Clin Oncol. 2017; 35(5): 544–551.
- Locke FL, Miklos D, Jacobson C, et al. Axicabtagene ciloleucel as second-line therapy for large B-cell lymphoma. N Engl J Med. 2022; 386(7): 640–654.
- Kamdar M, Solomon SR, Arnason J, et al. TRANSFORM Investigators. Lisocabtagene maraleucel versus standard of care with salvage chemotherapy followed by autologous stem cell transplantation as second-line treatment in patients with relapsed or refractory large B-cell lymphoma (TRANSFORM): results from an interim analysis of an open-label, randomised, phase 3 trial. Lancet. 2022; 399(10343): 2294–2308.
- Ghobadi A, Munoz J, Westin JR, et al. ZUMA-7 Investigators, Kite Members, All ZUMA-7 Investigators and Contributing Kite Members. Axicabtagene ciloleucel CAR t-cell therapy in refractory large b-cell lymphoma. N Engl J Med. 2017; 377(26): 2531–2544.
- Carlo-Stella C, Dickinson MJ, Iacoboni G, et al. Glofitamab, a novel, bivalent cd20-targeting t-cell-engaging bispecific antibody, induces durable complete remissions in relapsed or refractory b-cell lymphoma: a phase i trial. J Clin Oncol. 2021; 39(18): 1959–1970.
- Thieblemont C, Phillips T, Ghesquieres H, et al. Epcoritamab, a novel, subcutaneous CD3xCD20 bispecific t-cell-engaging antibody, in relapsed or refractory large b-cell lymphoma: dose expansion in a phase I/II trial. J Clin Oncol. 2023; 41(12): 2238–2247.
- Dreyling M, Goy A, Hess G, et al. Long-term outcomes with ibrutinib treatment for patients with relapsed/refractory mantle cell lymphoma: a pooled analysis of 3 clinical trials with nearly 10 years of follow-up. Hemasphere. 2022; 6(5): e712.
- Song Y, Zhou K, Zou D, et al. Zanubrutinib in relapsed/refractory mantle cell lymphoma: long-term efficacy and safety results from a phase 2 study. Blood. 2022; 139(21): 3148–3158.
- Wang ML, Jurczak W, Zinzani PL, et al. Pirtobrutinib in covalent bruton tyrosine kinase inhibitor pretreated mantle-cell lymphoma. J Clin Oncol. 2023; 41(24): 3988–3997.
- Moreau P, Kumar SK, San MJ, et al. Treatment of relapsed and refractory multiple myeloma: recommendations from the International Myeloma Working Group. Lancet Oncol. 2021; 22(3): e105–e118.
- Usmani SZ, Quach H, Mateos MV, et al. Carfilzomib, dexamethasone, and daratumumab versus carfilzomib and dexamethasone for patients with relapsed or refractory multiple myeloma (CANDOR): updated outcomes from a randomised, multicentre, open-label, phase 3 study. Lancet Oncol. 2022; 23(1): 65–76.
- Moreau P, Garfall A, Donk Nv, et al. Teclistamab in Relapsed or Refractory Multiple Myeloma. N Eng J Med. 2022; 387(6): 495–505.
- Tomasson MH, Iida S, Niesvizky R, et al. Elranatamab in relapsed or refractory multiple myeloma: phase 2 MagnetisMM-3 trial results. Nat Med. 2023; 29(9): 2259–2267.
- Chari A, Minnema MC, Berdeja JG, et al. Talquetamab, a t-cell-redirecting GPRC5D bispecific antibody for multiple myeloma. N Engl J Med. 2022; 387(24): 2232–2244.
- Dytfeld D, Wróbel T, Jamroziak K, et al. Carfilzomib, lenalidomide, and dexamethasone or lenalidomide alone as maintenance therapy after autologous stem-cell transplantation in patients with multiple myeloma (ATLAS): interim analysis of a randomised, open-label, phase 3 trial. Lancet Oncol. 2023; 24(2): 139–150.
- Erratum: second revision of the international staging system (R2-ISS) for overall survival in multiple myeloma: a European Myeloma Network (EMN) report within the HARMONY project. J Clin Oncol. 2022; 40(34): 4032.
- Moreau P, Masszi T, Grzasko N, et al. Oral ixazomib, lenalidomide, and dexamethasone for multiple myeloma. N Engl J Med. 2016; 374(17): 1621–1634.
- Newland A, Lee EJ, McDonald V, et al. Fostamatinib for persistent/chronic adult immune thrombocytopenia. Immunotherapy. 2018; 10(1): 9–25.
- Bussel JB, Arnold DM, Boxer MA, et al. Long-term fostamatinib treatment of adults with immune thrombocytopenia during the phase 3 clinical trial program. Am J Hematol. 2019; 94(5): 546–553.
- Dutt T, Shaw RJ, Stubbs M, et al. Real-world experience with caplacizumab in the management of acute TTP. Blood. 2021; 137(13): 1731–1740.
- Izuierdo CP, Mingot-Castellano ME, Fuentes AE, et al. And the Spanish thrombotic thrombocytopenic purpura registry (REPTT): real-world effectiveness of caplacizumab vs the standard of care in immune thrombotic thrombocytopenic purpura. Blood Adv. 2022; 5: 6219–6227.
- Srivastava A, Santagostino E, Dougall A, et al. WFH Guidelines for the Management of Hemophilia panelists and co-authors. WFH Guidelines for the Management of Hemophilia, 3rd edition. Haemophilia. 2020; 26 Suppl 6: 1–158.
- De la Corte-Rodriguez H, Rodriguez-Merchan EC. The role of physical medicine and rehabilitation in haemophiliac patients. Blood Coag Fibrinol. 2013; 24(1): 1–9.
- Kenet G, Chen YC, Lowe G, et al. Real-World rates of bleeding, factor VIII use, and quality of life in individuals with severe haemophilia a receiving prophylaxis in a prospective, noninterventional study. J Clin Med. 2021; 10(24).
- Marty FM, Ljungman PT, Chemaly RF, et al. Letermovir prophylaxis for cytomegalovirus in hematopoietic-cell transplantation. N Engl J Med. 2017; 377(25): 2433–2444.
- Russo D, Schmitt M, Pilorge S, et al. Efficacy and safety of extended duration letermovir prophylaxis in recipients of haematopoietic stem-cell transplantation at risk of cytomegalovirus infection: a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Haematol. 2024; 11(2): e127–e135.
- Avery RK, Alain S, Alexander BD, et al. SOLSTICE Trial Investigators. Maribavir for refractory cytomegalovirus infections with or without resistance post-transplant: results from a phase 3 randomized clinical trial. Clin Infect Dis. 2022; 75(4): 690–701.
- Maertens JA, Raad II, Marr KA, et al. Isavuconazole versus voriconazole for primary treatment of invasive mould disease caused by Aspergillus and other filamentous fungi (SECURE): a phase 3, randomised-controlled, non-inferiority trial. Lancet. 2016; 387(10020): 760–769.
- Richardson PG, Elias AD, Krishnan A, et al. Treatment of severe veno-occlusive disease with defibrotide: compassionate use results in response without significant toxicity in a high-risk population. Blood. 1998; 92(3): 737–744.
- Abecasis MM, Conceição Silva JP, Ferreira I, et al. Defibrotide as salvage therapy for refractory veno-occlusive disease of the liver complicating allogeneic bone marrow transplantation. Bone Marrow Transplant. 1999; 23(8): 843–846.
- Mahadeo K, Baiocchi R, Beitinjaneh A, et al. Tabelecleucel for allogeneic haematopoietic stem-cell or solid organ transplant recipients with Epstein–Barr virus-positive post-transplant lymphoproliferative disease after failure of rituximab or rituximab and chemotherapy (ALLELE): a phase 3, multicentre, open-label trial. Lancet Oncol. 2024; 25(3): 376–387.
