TY - JOUR
T1 - Clinical outcomes in adult patients with aplastic anemia
T2 - A single institution experience
AU - Boddu, Prajwal
AU - Garcia-Manero, Guillermo
AU - Ravandi, Farhad
AU - Borthakur, Gautam
AU - Jabbour, Elias
AU - DiNardo, Courtney
AU - Jain, Nitin
AU - Daver, Naval
AU - Pemmaraju, Naveen
AU - Anderlini, Paolo
AU - Parmar, Simrit
AU - Devendra, K. C.
AU - Akosile, Mary
AU - Pierce, Sherry A.
AU - Champlin, Richard
AU - Cortes, Jorge
AU - Kantarjian, Hagop
AU - Kadia, Tapan
N1 - Publisher Copyright:
© 2017 Wiley Periodicals, Inc.
PY - 2017/12
Y1 - 2017/12
N2 - Newer treatment modalities are being investigated to improve upon historical outcomes with standard immunosuppressive therapy (IST) in aplastic anemia (AA). We analyzed outcomes of adult patients with AA treated with various combinatorial anti-thymoglobulin-based IST regimens in frontline and relapsed/refractory (R/R) settings. Pretreatment and on-treatment clinical characteristics were analyzed for relationships to response and outcome. Among 126 patients reviewed, 95 were treatment-naïve (TN) and 63, R/R (including 32 from the TN cohort); median ages were 49 and 50 years, respectively. Overall survival (OS) was superior in IST responders (P <.001). Partial response to IST was associated with shorter relapse-free survival (RFS), as compared with complete response (P =.03). By multivariate analysis, baseline platelet and lymphocyte count predicted for IST response at 3 and 6 months, respectively. While additional growth factor interventions led to faster count recovery, there were no statistically significant differences in RFS or OS across the various frontline IST regimens (i.e., with/without G-CSF or eltrombopag). While marrow cellularity did not correlate with peripheral-blood counts at 3 months, cytomorphological assessment revealed dyspoietic changes in all nonresponders with hypercellular-marrow indices. Covert dysplasia, identified through early bone marrow assessment, has implications on future therapy choices after IST failure. Salvage IST response depended upon prior response to ATG: prior responders (46%) vs. primary refractory (0%) (P <.01). In the R/R setting, there was no survival difference between IST and allogeneic stem cell transplant groups, with a trend toward superior OS in the former. Transplant benefits in the R/R setting may be underrealized due to transplant-related mortality.
AB - Newer treatment modalities are being investigated to improve upon historical outcomes with standard immunosuppressive therapy (IST) in aplastic anemia (AA). We analyzed outcomes of adult patients with AA treated with various combinatorial anti-thymoglobulin-based IST regimens in frontline and relapsed/refractory (R/R) settings. Pretreatment and on-treatment clinical characteristics were analyzed for relationships to response and outcome. Among 126 patients reviewed, 95 were treatment-naïve (TN) and 63, R/R (including 32 from the TN cohort); median ages were 49 and 50 years, respectively. Overall survival (OS) was superior in IST responders (P <.001). Partial response to IST was associated with shorter relapse-free survival (RFS), as compared with complete response (P =.03). By multivariate analysis, baseline platelet and lymphocyte count predicted for IST response at 3 and 6 months, respectively. While additional growth factor interventions led to faster count recovery, there were no statistically significant differences in RFS or OS across the various frontline IST regimens (i.e., with/without G-CSF or eltrombopag). While marrow cellularity did not correlate with peripheral-blood counts at 3 months, cytomorphological assessment revealed dyspoietic changes in all nonresponders with hypercellular-marrow indices. Covert dysplasia, identified through early bone marrow assessment, has implications on future therapy choices after IST failure. Salvage IST response depended upon prior response to ATG: prior responders (46%) vs. primary refractory (0%) (P <.01). In the R/R setting, there was no survival difference between IST and allogeneic stem cell transplant groups, with a trend toward superior OS in the former. Transplant benefits in the R/R setting may be underrealized due to transplant-related mortality.
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U2 - 10.1002/ajh.24897
DO - 10.1002/ajh.24897
M3 - Article
C2 - 28850699
AN - SCOPUS:85030250815
SN - 0361-8609
VL - 92
SP - 1295
EP - 1302
JO - American Journal of Hematology
JF - American Journal of Hematology
IS - 12
ER -