Clinical characteristics and outcomes of 100 adult patients with pure red cell aplasia.
Cyclosporine A
Large granular lymphocytic leukemia
Pure red cell aplasia
Secondary
Journal
Annals of hematology
ISSN: 1432-0584
Titre abrégé: Ann Hematol
Pays: Germany
ID NLM: 9107334
Informations de publication
Date de publication:
Jul 2022
Jul 2022
Historique:
received:
21
11
2021
accepted:
17
04
2022
pubmed:
24
4
2022
medline:
22
6
2022
entrez:
23
4
2022
Statut:
ppublish
Résumé
Adult pure red cell aplasia (PRCA) is a rare syndrome characterized by a severe normocytic anemia, reticulocytopenia, and absence of erythroblasts from bone marrow. The standard treatment has not yet been established for PRCA, although cyclosporine (CsA), corticosteroids (CS) showed a response in PRCA. We retrospectively analyzed the clinical data of 60 primary and 40 secondary adult patients with acquired PRCA. The proportion of secondary PRCA is relatively high and commonly associated with large granular lymphocyte leukemia (LGLL) (28 cases, 70.0%). The remission-induced regimens included CS, CsA, or other agents, and the response rate was 66.7%, 71.4%, and 50%, respectively (P = 0.336). When treating with CsA, the response rate of LGLL-associated PRCA was lower than primary PRCA (42.1% vs 85.7%, P = 0.001). Logistic regression analysis showed that ORR was inversely related to LGLL-associated PRCA. LGLL-associated PRCA had poor therapeutic efficacy to CsA.
Identifiants
pubmed: 35460389
doi: 10.1007/s00277-022-04847-2
pii: 10.1007/s00277-022-04847-2
doi:
Substances chimiques
Adrenal Cortex Hormones
0
Cyclosporine
83HN0GTJ6D
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
1493-1498Subventions
Organisme : State Administration of Traditional Chinese Medicine Industry Specialty
ID : 201407001-4
Organisme : National Public Health Grand Research Foundation
ID : 201202017
Organisme : Priority Academic Program Development of Jiangsu Higher Education Institute
ID : JX10231801
Organisme : Jiangsu Provincial Special Program of Medical Science
ID : BL201408
Informations de copyright
© 2022. The Author(s), under exclusive licence to Springer-Verlag GmbH Germany, part of Springer Nature.
Références
Means RT (2016) Pure red cell aplasia. Blood 128(21):2504–2509. https://doi.org/10.1182/blood-2016-05-717140
doi: 10.1182/blood-2016-05-717140
pubmed: 27881371
Lacy M (1996) Pure red cell aplasia : association with large granular lymphocyte leukemia and the prognostic value of cytogenetic abnormalities. Blood 87(7):3000
doi: 10.1182/blood.V87.7.3000.bloodjournal8773000
Dessypris E (1991) The biology of pure red cell aplasia. Semin Hematol 28(4):275–284
pubmed: 1759168
Thompson CA, Steensma DP (2006) Pure red cell aplasia associated with thymoma: clinical insights from a 50-year single-institution experience. Br J Haematol 135(3):405–407. https://doi.org/10.1111/j.1365-2141.2006.06295.x
doi: 10.1111/j.1365-2141.2006.06295.x
pubmed: 17032177
Zecca M, Stefano PD, Nobili B, Locatelli F (2001) Anti-CD20 monoclonal antibody for the treatment of severe, immune-mediated, pure red cell aplasia and hemolytic anemia. Blood 97(12):3995–3997. https://doi.org/10.1182/blood.v97.12.3995
doi: 10.1182/blood.v97.12.3995
pubmed: 11389047
Risitano AM, Selleri C, Serio B, Torelli GF, Kulagin A, Maury S, Halter J, Gupta V, Bacigalupo A, Sociè G, Tichelli A, Schrezenmeier H, Marsh J, Passweg J, Rotoli B, on behalf of the Working Party Severe Aplastic Anaemia (WPSAA) of the European Group for Blood and Marrow Transplantation (EBMT) (2010) Alemtuzumab is safe and effective as immunosuppressive treatment for aplastic anaemia and single-lineage marrow failure: a pilot study and a survey from the EBMT WPSAA. Br J Haematol 148(5):791–796. https://doi.org/10.1111/j.1365-2141.2009.08027.x
doi: 10.1111/j.1365-2141.2009.08027.x
pubmed: 19995389
Sloand EM, Olnes MJ, Weinstein B, Wu C, Maciejewski J, Scheinberg P, Young NS (2010) Long-term follow-up of patients with moderate aplastic anemia and pure red cell aplasia treated with daclizumab. Haematologica 95(3):382–387. https://doi.org/10.3324/haematol.2009.013557
doi: 10.3324/haematol.2009.013557
pubmed: 20207845
pmcid: 2833067
Gupta R, Ezeonyeji A, Thomas A, Scully M, Ehrenstein M, Isenberg D (2011) A case of pure red cell aplasia and immune thrombocytopenia complicating systemic lupus erythematosus: Responseto rituximab and cyclophosphamide. Lupus 20(14):1547–1550. https://doi.org/10.1177/0961203311411349
doi: 10.1177/0961203311411349
pubmed: 21993386
Rossignol J, Michallet AS, Oberic L, Picard M, Garon A, Willekens C, Dulery R, Leleu X, Cazin B, Ysebaert L (2010) Rituximab-cyclophosphamide-dexamethasone combination in the management of autoimmune cytopenias associated with chronic lymphocytic leukemia. Leukemia 25(3):473–478. https://doi.org/10.1038/leu.2010.278
doi: 10.1038/leu.2010.278
pubmed: 21127498
Sawada K, Hirokawa M, Fujishima N, TeramuraM Bessho M, Dan K, Tsurumi H, Nakao S, Urabe A, Omine M, Ozawa K, PRCA Collaborative Study Group (2007) Long-term outcome of patients with acquired primary idiopathic pure red cell aplasia receiving cyclosporine A. A nationwide cohort study in Japan for the PRCA Collaborative Study Group. Haematologica 92(8):1021–1028. https://doi.org/10.3324/haematol.11192
doi: 10.3324/haematol.11192
pubmed: 17640861
Wu X, Wang S, Lu X, Shen W, Qiao C, Wu Y, Lu R, Wang S, Zhang J, Hong M, Zhu Y, Li J, He G (2018) Response to cyclosporine A and corticosteroids in adult patients with acquired pure red cell aplasia: serial experience at a single center. Int J Hematol 108(2):123–129. https://doi.org/10.1007/s12185-018-2446-y
doi: 10.1007/s12185-018-2446-y
pubmed: 29589280
Hirokawa M, Sawada K, Fujishima N, Nakao S, Urabe A, Dan K, Fujisawa S, Yonemura Y, Kawano F, Omine M, Ozawa K, PRCA Collaborative Study Group (2008) Long-term response and outcome following immunosuppressive therapy in thymoma associated pure red cell aplasia: a nationwide cohort study in Japan by the PRCA collaborative study group. Haematologica 93(1):27–33. https://doi.org/10.3324/haematol.11655
doi: 10.3324/haematol.11655
pubmed: 18166782
Swerdlow SH, Campo E, Harris NL, Jaffe ES, Pileri SA, Stein H et al (2017) WHO Classification of tumours of haematopoietic and lymphoid tissues, Revised 4th. IARC, Lyon, pp 348–354
Lamy T, Moignet A, Loughran TP Jr (2017) LGL leukemia: from pathogenesis to treatment. Blood 129(9):1082–1094. https://doi.org/10.1182/blood-2016-08-692590
doi: 10.1182/blood-2016-08-692590
pubmed: 28115367
Gurnari C, Maciejewski JP (2021) How I manage acquired pure red cell aplasia in adults. Blood 137(15):2001–2009. https://doi.org/10.1182/blood.2021010898
doi: 10.1182/blood.2021010898
pubmed: 33657207
pmcid: 8057257
Mangla A, Hamad H (2022) Pure Red Cell Aplasia. In: StatPearls. StatPearls Publishing, Treasure Island
Hirokawa M, Sawada K, Fujishima N, Teramura M, Bessho M, Dan K, Tsurumi H, Nakao S, Urabe A, Fujisawa S, Yonemura Y, Kawano F, Oshimi K, Sugimoto K, Matsuda A, Karasawa M, Arai A, Komatsu N, Harigae H, Omine M, Ozawa K, Kurokawa M, for the PRCA Collaborative Study Group (2015) Long-term outcome of patients with acquired chronic pure red cell aplasia (PRCA) following immunosuppressive therapy: a final report of the nationwide cohort study in 2004/2006 by the Japan PRCA collaborative study group. Br J Haematol 169(6):879–886. https://doi.org/10.1111/bjh.13376
doi: 10.1111/bjh.13376
pubmed: 25807974
Kawahara S, Sasaki M, Isobe Y, Ando J, Noguchi M, Koike M, Hirano T, Oshimi K, Sugimoto K (2009) Clinical analysis of 52 patients with granular lymphocyte proliferative disorder (GLPD) showed frequent anemia in indolent T-cell GLPD in Japan. Eur J Haematol 82(4):308–314. https://doi.org/10.1111/j.1600-0609.2009.01213.x
doi: 10.1111/j.1600-0609.2009.01213.x
pubmed: 19220421
Kwong YL, Au WY, Leung AY, Tse EW (2010) T-cell large granular lymphocyte leukemia: an Asian perspective. Ann Hematol 89(4):331–339. https://doi.org/10.1007/s00277-009-0895-3
doi: 10.1007/s00277-009-0895-3
pubmed: 20084380
pmcid: 7102052
Qiu ZY, Fan L, Wang L, Qiao C, Wu YJ, Zhou JF (2013) Xu W and Li JY (2006) STAT3 mutations are frequent in T-cell large granular lymphocytic leukemia with pure red cell aplasia. J Hematol Oncol 6(1):82. https://doi.org/10.1186/1756-8722-6-82
doi: 10.1186/1756-8722-6-82
pubmed: 24283217
pmcid: 4222121
Burks EJ, Loughran TP (2006) Pathogenesis of neutropenia in large granular lymphocyte leukemia and Felty syndrome. Blood Rev 20(5):245–266. https://doi.org/10.1016/j.blre.2006.01.003
doi: 10.1016/j.blre.2006.01.003
pubmed: 16530306
Liu X, Lu X, Chen L, Yang Y, Wu X, Lu R, Wang S, Zhang J, Hong M, Zhu Y, He G, Li J (2020) Immunosuppressive therapy for elderly-acquired pure red cell aplasia: cyclosporine A may be more effective. Ann Hematol 99(3):443–449. https://doi.org/10.1007/s00277-020-03926-6
doi: 10.1007/s00277-020-03926-6
pubmed: 31970447
Dong N, Castillo Tokumori F, Isenalumhe L, Zhang Y, Tandon A, Knepper TC, Mo Q, Shao H, Zhang L, Sokol L (2021) Large granular lymphocytic leukemia–A retrospective study of 319 cases. Am J Hematol 96(7):772–780. https://doi.org/10.1002/ajh.26183
doi: 10.1002/ajh.26183
pubmed: 33819354
Fujishima N, Sawada K, Hirokawa M, Oshimi K, Sugimoto K, Matsuda A, Teramura M, Karasawa M, Arai A, Yonemura Y, Nakao S, Urabe A, Omine M, Ozawa K, PRCA Collaborative Study Group (2008) Long-term responses and outcomes following immunosuppressive therapy in large granular lymphocyte leukemia-associated pure red cell aplasia: a Nationwide Cohort Study in Japan for the PRCA Collaborative Study Group. Haematologica 93(10):1555–1559. https://doi.org/10.3324/haematol.12871
doi: 10.3324/haematol.12871
pubmed: 18641028
Ishida F, Matsuda K, Sekiguchi N, Makishima H, Taira C, Momose K, Nishina S, Senoo N, Sakai H, Ito T, Kwong YL (2014) STAT3 gene mutations and their association with pure red cell aplasia in large granular lymphocyte Leukemia. Cancer Sci 105(3):342–346. https://doi.org/10.1111/cas.12341
doi: 10.1111/cas.12341
pubmed: 24350896
pmcid: 4317942
Liu X, Cheng L, He Y, Zhang R, Lu R, Zhang J, Hong M, He G, Li J (2021) Eltrombopag restores erythropoiesis in refractory adult acquired pure red cell aplasia. Int J Hematol 114(1):124–128. https://doi.org/10.1007/s12185-021-03100-2
doi: 10.1007/s12185-021-03100-2
pubmed: 33611753
Jiang H, Zhang H, Wang H, Qi W, Cao Q, Xing L, Fu R, Shao Z, Wang H (2019) Sirolimus for the treatment of multi-resistant pure red cell Aplasia. Br J Haematol 184(6):1055–1058. https://doi.org/10.1111/bjh.15245
doi: 10.1111/bjh.15245
pubmed: 29741762