PMID- 33855784
OWN - NLM
STAT- MEDLINE
DCOM- 20220304
LR  - 20220304
IS  - 1545-5017 (Electronic)
IS  - 1545-5009 (Linking)
VI  - 68
IP  - 8
DP  - 2021 Aug
TI  - Eltrombopag in children with severe aplastic anemia.
PG  - e29066
LID - 10.1002/pbc.29066 [doi]
AB  - BACKGROUND: Immunosuppressive therapy with horse antithymocyte globulin and 
      cyclosporine currently remains the standard therapy for children with severe 
      aplastic anemia (SAA) who lack human leukocyte antigen (HLA)-identical sibling. 
      The thrombopoietin receptor agonist eltrombopag has been recently approved for 
      SAA patients 2 years and older. However, there are limited data on its safety and 
      efficacy in pediatric cohorts. METHODS: We conducted a retrospective study of 
      patients </=18 years old consecutively diagnosed with SAA between 2000 and 2018. 
      Patients received either standard immunosuppressive therapy (IST-Std) or IST with 
      eltrombopag (IST-Epag). The primary outcome was the objective response (OR), 
      including partial and complete response (CR), at 6 and 12 months after starting 
      therapy. RESULTS: We identified 16 patients receiving IST-Std and nine IST-Epag 
      treatment (seven of nine as upfront therapy and two of seven after previously 
      failed IST). The OR at 6 and 12 months in IST-Std arm was 71% and 100%, with CR 
      in 29% and 58%, respectively. Seven patients receiving upfront IST-Epag had OR at 
      6 and 12 months, with two of seven (29%) achieving CR at 6 and 12 months. Two 
      patients who previously failed standard IST did not respond to eltrombopag. No 
      significant differences were observed in both cohorts with regard to infections. 
      One IST-Epag-treated patient developed transient grade 3 transaminitis. Finally, 
      no changes in paroxysmal nocturnal hemoglobinuria (PNH) clone size and 
      cytogenetic abnormalities were seen in either cohort. CONCLUSION: The addition of 
      eltrombopag to standard IST was well tolerated and resulted in satisfactory 
      hematological response at 6 and 12 months in this single-institution experience. 
      A larger cohort with longer follow-up is required to assess response durability.
CI  - (c) 2021 Wiley Periodicals LLC.
FAU - Lesmana, Harry
AU  - Lesmana H
AUID- ORCID: 0000-0001-5496-3839
AD  - Department of Hematology, St. Jude Children's Research Hospital, Memphis, 
      Tennessee, USA.
AD  - Department of Pediatric Hematology, Oncology and Bone Marrow Transplantation, 
      Cleveland Clinic, Cleveland, Ohio, USA.
FAU - Jacobs, Timothy
AU  - Jacobs T
AD  - Department of Pharmaceutical Sciences, St. Jude Children's Research Hospital, 
      Memphis, Tennessee, USA.
FAU - Boals, Michelle
AU  - Boals M
AD  - Department of Hematology, St. Jude Children's Research Hospital, Memphis, 
      Tennessee, USA.
FAU - Gray, Nathan
AU  - Gray N
AD  - Department of Hematology, St. Jude Children's Research Hospital, Memphis, 
      Tennessee, USA.
FAU - Lewis, Sara
AU  - Lewis S
AD  - Department of Hematology, St. Jude Children's Research Hospital, Memphis, 
      Tennessee, USA.
FAU - Ding, Juan
AU  - Ding J
AD  - Department of Biostatistics, St. Jude Children's Research Hospital, Memphis, 
      Tennessee, USA.
FAU - Kang, Guolian
AU  - Kang G
AD  - Department of Biostatistics, St. Jude Children's Research Hospital, Memphis, 
      Tennessee, USA.
FAU - Hale, Melvanique
AU  - Hale M
AD  - Department of Hematology, St. Jude Children's Research Hospital, Memphis, 
      Tennessee, USA.
FAU - Weiss, Mitchell
AU  - Weiss M
AD  - Department of Hematology, St. Jude Children's Research Hospital, Memphis, 
      Tennessee, USA.
FAU - Reiss, Ulrike
AU  - Reiss U
AD  - Department of Hematology, St. Jude Children's Research Hospital, Memphis, 
      Tennessee, USA.
FAU - Wang, Winfred
AU  - Wang W
AUID- ORCID: 0000-0001-6774-9698
AD  - Department of Hematology, St. Jude Children's Research Hospital, Memphis, 
      Tennessee, USA.
FAU - Wlodarski, Marcin
AU  - Wlodarski M
AD  - Department of Hematology, St. Jude Children's Research Hospital, Memphis, 
      Tennessee, USA.
LA  - eng
PT  - Journal Article
DEP - 20210415
PL  - United States
TA  - Pediatr Blood Cancer
JT  - Pediatric blood & cancer
JID - 101186624
RN  - 0 (Antilymphocyte Serum)
RN  - 0 (Benzoates)
RN  - 0 (Hydrazines)
RN  - 0 (Immunosuppressive Agents)
RN  - 0 (Pyrazoles)
RN  - 83HN0GTJ6D (Cyclosporine)
RN  - S56D65XJ9G (eltrombopag)
SB  - IM
MH  - *Anemia, Aplastic/drug therapy
MH  - Antilymphocyte Serum
MH  - Benzoates/*therapeutic use
MH  - Child
MH  - Cyclosporine
MH  - Humans
MH  - Hydrazines/*therapeutic use
MH  - Immunosuppressive Agents/*therapeutic use
MH  - Pyrazoles/*therapeutic use
MH  - Retrospective Studies
MH  - Treatment Outcome
OTO - NOTNLM
OT  - aplastic anemia
OT  - bone marrow failure
OT  - eltrombopag
EDAT- 2021/04/16 06:00
MHDA- 2022/03/05 06:00
CRDT- 2021/04/15 07:46
PHST- 2021/03/04 00:00 [revised]
PHST- 2020/11/06 00:00 [received]
PHST- 2021/03/22 00:00 [accepted]
PHST- 2021/04/16 06:00 [pubmed]
PHST- 2022/03/05 06:00 [medline]
PHST- 2021/04/15 07:46 [entrez]
AID - 10.1002/pbc.29066 [doi]
PST - ppublish
SO  - Pediatr Blood Cancer. 2021 Aug;68(8):e29066. doi: 10.1002/pbc.29066. Epub 2021 
      Apr 15.