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denotes that this is a ticketed session.Type: Oral
Session: 615. Acute Myeloid Leukemias: Clinical and Epidemiological: Treatments and Outcomes in AML in Specific Age Groups, and in Blastic Plasmacytoid Dendritic Cell Neoplasms
Hematology Disease Topics & Pathways:
Acute Myeloid Malignancies, AML, Combination therapy, Adult, Research, Epidemiology, Clinical Practice (Health Services and Quality), Clinical Research, Education, Diseases, Treatment Considerations, Clinical procedures, Myeloid Malignancies, Technology and Procedures, Study Population, Human
Material: This multinational retrospective study performed by PALG and PETHEMA (EPI-BLAS study) groups registered BPDCN patients (WHO 2008 or 2016) from June 1999, onwards. We categorized patients into 3 groups based on the therapeutic approaches: intensive treatment, non-intensive treatment, and supportive care only treatment. We analyzed composite complete remission (CRc) after first-line and overall survival (OS). CRc was defined as the sum of full CR, clinical CR with cutaneous sequelae, clinical CR with hematologic partial response, and clinical CR with incomplete recovery.
Results: Overall, 232 BPDCN pts were registered among 21866 AML cases in the PETHEMA registry (0.11%), and 228 were evaluable. Most were male (77%), median age was 63 years old (range 17-90), and 45 (26%) had prior or concomitant neoplastic disease (53% of them had hematological neoplasms, including large granular lymphocytic leukemia (n=1), myelodysplastic syndromes (n=11), non-Hodgkin T-cell lymphoma (n=3), and chronic myelomonocytic leukemia(n=6)). Median hemoglobin level was 10.55 g/dL (range 5.5-16.8), white blood cell count was 6.55x 10^3/mL (0.1-163), platelet count was 94 x10^3/µL (6-457), peripheral blood blasts 18.5% (0-95), bone marrow blasts 54% (0-98), LDH 331.5 IU/L (111-4500), creatinine 0.9mg/dL (0.4- 8.2), uric acid level 5.2mg/dL (2.2- 13.8). As for extramedullary involvement,161 (80%) out of 202 with available data, had skin involvement, hepato or splenomegaly was found in 21%, 49% had lymphadenopathy and 80% presented with bone marrow affection. A lumbar puncture was performed at diagnosis in 110 (65%) patients of the 170 patients with available data; of them, 33 (30%) had infiltration. As per available immunohistochemistry/phenotype markers, CD123, CD56 and CD4 were positive in 160 (92%), 154 (83%), and 157 (87%) pts, respectively. First-line approach was available in 200 patients, 15 (7%) received supportive care; 132 (59%) intensive; and 53 (22%) non-intensive treatment. Among intensive group, 45 (34%) received AML-like (34 with 3+7, 9 FLAG-IDA, and 2 HAM); 60 (46%) ALL-like (55 HyperCVAD and 5 with PETHEMA-ALL), and 27 (20%) lymphoma-like (17 CHOP, 7 etoposide-based, 1 ABVD, and 2 bortezomib-based). The non-intensive group included 21 (40%) AML-like regimens (5 AZA-VEN, 2 FLUGA, 10 AZA, and 4 LDAC); 18 (34%) CD123 or CD33 targeted molecules, 9 (16%) ALL-like, 5 (10%) lymphoma-like, including 3 with radiotherapy or intralesional chemotherapy. Among 185 pts with active treatment, response was available in 162 pts, of whom 117 (72%) obtained CRc, while 14 pts (9%) died during induction (sepsis [n=8], multiorgan failure [n=3], bleeding [n=2], and pulmonary embolism [n=1]). Median OS of the entire cohort was 11.83 months [95% CI 08.76 – 14.90]. Median OS for patients who achieved CRc after first-line therapy was 27.47 months (95% CI: 15.08–39.85) and 7.03 months (95% CI: 3.79-10.28) for those not achieving CRc; p<0.001. A total of 65 pts (55% of those in first CRc) were transplanted in first CRc (61 an allogeneic transplant, and 4 an autologous one). Median OS in patients who received a transplantation was 84.76 (95% CI 0.00– 173.49) months and in patients not transplanted was 14.80 (95% CI 10.22-19.38) months (p<0.001).
Conclusion: Clinical and biological features were consistent with previous reports, although skin infiltration and CD123 expression were below expected. We show heterogeneity in therapeutic approaches, relatively high rate of CRc after front-line, and a sizable proportion of transplanted patients. However, median OS remains disappointing. Improvement of outcomes through well-defined treatment protocols and targeted agents is needed for this high unmet need disease.
Disclosures: The EPI-BLAs registry was partially funded by Menarini.
Disclosures: Rodriguez Macias: Astellas: Consultancy, Honoraria, Speakers Bureau; Jazz Pharmaceuticals: Consultancy, Honoraria, Speakers Bureau; Servier: Consultancy, Honoraria, Speakers Bureau; Otsuka: Consultancy, Honoraria, Speakers Bureau; BMS-Celgene: Consultancy, Honoraria, Speakers Bureau; Abbvie: Consultancy, Honoraria, Speakers Bureau. Tormo: Janssen, AbbVie, Jazz: Other: Travel grant for attending meetings; AbbVie, Gilead, Pfizer, Astellas, BMS: Honoraria; SOBI: Other: Data Safety Monitoring Board. Montesinos: Glycomimetics: Consultancy; Servier: Consultancy, Membership on an entity's Board of Directors or advisory committees, Other: research support, Research Funding, Speakers Bureau; Jazzpharma: Consultancy, Research Funding, Speakers Bureau; Janssen: Membership on an entity's Board of Directors or advisory committees, Other: research support, Speakers Bureau; Pfizer: Consultancy, Research Funding, Speakers Bureau; Daiichi Sankyo, Inc.: Consultancy, Membership on an entity's Board of Directors or advisory committees, Other: research support, Research Funding, Speakers Bureau; Astellas: Consultancy, Membership on an entity's Board of Directors or advisory committees, Other: research support, Speakers Bureau; AbbVie: Consultancy, Membership on an entity's Board of Directors or advisory committees, Other: research support, Research Funding, Speakers Bureau; Novartis: Consultancy, Research Funding, Speakers Bureau; Kura Oncology: Consultancy; Syndax: Consultancy.
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