Effective Tacrolimus Treatment for Patients with Non-Severe Aplastic Anemia That is Refractory/Intolerant to Cyclosporine A: A Retrospective Study.


Journal

Drug design, development and therapy
ISSN: 1177-8881
Titre abrégé: Drug Des Devel Ther
Pays: New Zealand
ID NLM: 101475745

Informations de publication

Date de publication:
2020
Historique:
received: 18 08 2020
accepted: 10 11 2020
entrez: 7 1 2021
pubmed: 8 1 2021
medline: 22 9 2021
Statut: epublish

Résumé

For symptomatic non-severe aplastic anemia (NSAA) patients who cannot afford anti-thymocyte globulin (ATG) or allogeneic hematopoietic stem cell transplantation (HSCT), tacrolimus (FK) may be an option if these patients do not respond or become tolerant to cyclosporine A (CsA). We enrolled 101 NSAA patients who were refractory or intolerant to CsA with no chance of HSCT or ATG treatment and treated these patients with tacrolimus for at least 6 months, with follow-up for at least one year. The overall response rate (ORR) was 38.6% (complete response: 9.9%; partial response: 28.7%), and the median time to optimal response was 6 (3~10) months. Thirty-two (31.7%) cases had elevated creatinine levels. Eight (7.9%) cases had elevations in AST/ALT. A total of 25.6% (10/39) of patients relapsed at the end of follow-up. Age ( Our data suggest that tacrolimus is a salvage treatment for patients with NSAA that is refractory or intolerant to CsA.

Sections du résumé

BACKGROUND BACKGROUND
For symptomatic non-severe aplastic anemia (NSAA) patients who cannot afford anti-thymocyte globulin (ATG) or allogeneic hematopoietic stem cell transplantation (HSCT), tacrolimus (FK) may be an option if these patients do not respond or become tolerant to cyclosporine A (CsA).
METHODS METHODS
We enrolled 101 NSAA patients who were refractory or intolerant to CsA with no chance of HSCT or ATG treatment and treated these patients with tacrolimus for at least 6 months, with follow-up for at least one year.
RESULTS RESULTS
The overall response rate (ORR) was 38.6% (complete response: 9.9%; partial response: 28.7%), and the median time to optimal response was 6 (3~10) months. Thirty-two (31.7%) cases had elevated creatinine levels. Eight (7.9%) cases had elevations in AST/ALT. A total of 25.6% (10/39) of patients relapsed at the end of follow-up. Age (
CONCLUSION CONCLUSIONS
Our data suggest that tacrolimus is a salvage treatment for patients with NSAA that is refractory or intolerant to CsA.

Identifiants

pubmed: 33408460
doi: 10.2147/DDDT.S275975
pii: 275975
pmc: PMC7779311
doi:

Substances chimiques

Immunosuppressive Agents 0
Cyclosporine 83HN0GTJ6D
Tacrolimus WM0HAQ4WNM

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

5711-5719

Informations de copyright

© 2020 Du et al.

Déclaration de conflit d'intérêts

The authors declare that they have no conflicts of interest for this work.

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Auteurs

Yali Du (Y)

Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, People's Republic of China.

Yuzhou Huang (Y)

Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, People's Republic of China.

Wenzhe Zhou (W)

Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, People's Republic of China.

Xinjian Liu (X)

Department of Hematology, Affiliated Cancer Hospital of Zhengzhou University, Henan Cancer Hospital, Institute of Hematology of Henan Province, Zhengzhou, People's Republic of China.

Fangfei Chen (F)

Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, People's Republic of China.

Chen Yang (C)

Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, People's Republic of China.

Miao Chen (M)

Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, People's Republic of China.

Jing Ruan (J)

Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, People's Republic of China.

Bing Han (B)

Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, People's Republic of China.

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Classifications MeSH