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Refractory Thrombocytopenia

MedGen UID:
443195
•Concept ID:
C2826321
•
Neoplastic Process
Synonym: Refractory thrombocytopenia
SNOMED CT: Refractory thrombocytopenia (721304007)

Definition

A myelodysplastic syndrome characterized by the presence of at least 10% dysplastic megakaryocytes, found within at least 30 megakaryocytes examined in the bone marrow. [from NCI]

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • CROGVRefractory Thrombocytopenia

Professional guidelines

PubMed

Improved detection of sinusoidal obstructive syndrome using pediatric-AYA diagnostic criteria and severity grading.
Ragoonanan D, Khazal SJ, Wang J, Payne A, Kohorst M, Harden A, Tewari P, Petropoulos D, Shoberu B, Kebriaei P, Mahadeo KM, Tambaro FP
Bone Marrow Transplant 2021 Jan;56(1):175-184. Epub 2020 Jul 14 doi: 10.1038/s41409-020-00998-w. PMID: 32665674
Refractory Thrombocytopenia Is a Valid Early Diagnostic Criteria for Hepatic Veno-Occlusive Disease in Children.
Embaby MM, Rangarajan HG, Abu-Arja R, Auletta JJ, Stanek J, Pai V, Nicol KK, Bajwa RS
Biol Blood Marrow Transplant 2020 Mar;26(3):546-552. Epub 2019 Nov 19 doi: 10.1016/j.bbmt.2019.11.012. PMID: 31756537
Danazol in the treatment of systemic lupus erythematosus: a qualitative systematic review.
Letchumanan P, Thumboo J
Semin Arthritis Rheum 2011 Feb;40(4):298-306. Epub 2010 Jun 11 doi: 10.1016/j.semarthrit.2010.03.005. PMID: 20541792

Recent clinical studies

Etiology

Predictors of refractory risk in systemic lupus erythematosus-related thrombocytopenia: a dual-centre retrospective study.
Su K, Cheng H, Jia Z, Yuan Y, Yang H, Gao Q, Jiang Z, Wen H, Jiang J
Lupus Sci Med 2022 May;9(1) doi: 10.1136/lupus-2022-000677. PMID: 35606019Free PMC Article
Eltrombopag improves refractory thrombocytopenia in patients with Sjögren's syndrome.
Xu L, Zhang Y, Lin N, Song X, Dai Q
Sci Prog 2022 Apr-Jun;105(2):368504221102786. doi: 10.1177/00368504221102786. PMID: 35603866Free PMC Article
Severe Thrombocytopenia in Patients With Advanced Neuroendocrine Tumor Treated With Peptide Receptor Radioligand Therapy.
Naveed Ahmad JA, Schroeder BB, Ruhoy SM, Kennecke HF, Lin BS
Clin Nucl Med 2022 May 1;47(5):409-413. doi: 10.1097/RLU.0000000000004130. PMID: 35307721Free PMC Article
Eltrombopag for the treatment of refractory thrombocytopenia associated with connective tissue disease.
Wang J, Dai M, Fu Q, Chen S
Sci Rep 2021 Mar 9;11(1):5459. doi: 10.1038/s41598-021-84493-2. PMID: 33750817Free PMC Article
Kaposiform lymphangiomatosis and kaposiform hemangioendothelioma: similarities and differences.
Ji Y, Chen S, Peng S, Xia C, Li L
Orphanet J Rare Dis 2019 Jul 5;14(1):165. doi: 10.1186/s13023-019-1147-9. PMID: 31277673Free PMC Article

Diagnosis

Low-dose decitabine for refractory thrombocytopenia following allogeneic hematopoietic stem cell transplantation in children: A pilot study.
Liu N, Zhao FY, Liang J, Shang LP, Xu XJ
Int Immunopharmacol 2023 Feb;115:109579. Epub 2022 Dec 26 doi: 10.1016/j.intimp.2022.109579. PMID: 36577152
Clinical variability in multifocal lymphangioendotheliomatosis with thrombocytopenia: a review of the literature.
Manor J, Patel K, Iacobas I, Margolin JF, Mahajan P
Pediatr Hematol Oncol 2021 May;38(4):367-377. Epub 2021 Feb 27 doi: 10.1080/08880018.2020.1871135. PMID: 33641614
Refractory Thrombocytopenia Is a Valid Early Diagnostic Criteria for Hepatic Veno-Occlusive Disease in Children.
Embaby MM, Rangarajan HG, Abu-Arja R, Auletta JJ, Stanek J, Pai V, Nicol KK, Bajwa RS
Biol Blood Marrow Transplant 2020 Mar;26(3):546-552. Epub 2019 Nov 19 doi: 10.1016/j.bbmt.2019.11.012. PMID: 31756537
Kaposiform lymphangiomatosis and kaposiform hemangioendothelioma: similarities and differences.
Ji Y, Chen S, Peng S, Xia C, Li L
Orphanet J Rare Dis 2019 Jul 5;14(1):165. doi: 10.1186/s13023-019-1147-9. PMID: 31277673Free PMC Article
Hunter syndrome with persistent thrombocytopenia.
Panigrahi I, Dhanorkar M, Didel S, Koganti RA
BMJ Case Rep 2019 Apr 20;12(4) doi: 10.1136/bcr-2018-226518. PMID: 31005860Free PMC Article

Therapy

Eltrombopag improves refractory thrombocytopenia in patients with Sjögren's syndrome.
Xu L, Zhang Y, Lin N, Song X, Dai Q
Sci Prog 2022 Apr-Jun;105(2):368504221102786. doi: 10.1177/00368504221102786. PMID: 35603866Free PMC Article
Low-dose rituximab therapy in steroid-refractory thrombocytopenia due to systemic lupus erythematosus.
Ilizaliturri-Guerra O, Uriarte-Botello R, Pineda-Sic RÁ, Serna-Peña G, Garza-Elizondo MA, Galarza-Delgado DÁ, Leal-Bramasco AS, Elizondo-Solís CV, Santoyo-Fexas L, Villarreal-Alarcón MÁ
Rheumatol Int 2020 Oct;40(10):1717-1724. Epub 2020 Aug 14 doi: 10.1007/s00296-020-04668-4. PMID: 32797277
Eltrombopag is an effective and safe therapy for refractory thrombocytopenia after haploidentical hematopoietic stem cell transplantation.
Fu H, Zhang X, Han T, Mo X, Wang Y, Chen H, Han W, Wang J, Wang F, Yan C, Zhang Y, Sun Y, Liu K, Huang X, Xu L
Bone Marrow Transplant 2019 Aug;54(8):1310-1318. Epub 2019 Jan 21 doi: 10.1038/s41409-019-0435-2. PMID: 30664724
Anti B-cell therapy against refractory thrombocytopenia in SLE and MCTD patients: long-term follow-up and review of the literature.
Jovancevic B, Lindholm C, Pullerits R
Lupus 2013 Jun;22(7):664-74. Epub 2013 Apr 23 doi: 10.1177/0961203313485489. PMID: 23612795
HIV-related thrombocytopenia.
Scaradavou A
Blood Rev 2002 Mar;16(1):73-6. doi: 10.1054/blre.2001.0188. PMID: 11914001

Prognosis

Predictors of refractory risk in systemic lupus erythematosus-related thrombocytopenia: a dual-centre retrospective study.
Su K, Cheng H, Jia Z, Yuan Y, Yang H, Gao Q, Jiang Z, Wen H, Jiang J
Lupus Sci Med 2022 May;9(1) doi: 10.1136/lupus-2022-000677. PMID: 35606019Free PMC Article
Eltrombopag improves refractory thrombocytopenia in patients with Sjögren's syndrome.
Xu L, Zhang Y, Lin N, Song X, Dai Q
Sci Prog 2022 Apr-Jun;105(2):368504221102786. doi: 10.1177/00368504221102786. PMID: 35603866Free PMC Article
Eltrombopag for the treatment of refractory thrombocytopenia associated with connective tissue disease.
Wang J, Dai M, Fu Q, Chen S
Sci Rep 2021 Mar 9;11(1):5459. doi: 10.1038/s41598-021-84493-2. PMID: 33750817Free PMC Article
Kaposiform lymphangiomatosis and kaposiform hemangioendothelioma: similarities and differences.
Ji Y, Chen S, Peng S, Xia C, Li L
Orphanet J Rare Dis 2019 Jul 5;14(1):165. doi: 10.1186/s13023-019-1147-9. PMID: 31277673Free PMC Article
HIV-related thrombocytopenia.
Scaradavou A
Blood Rev 2002 Mar;16(1):73-6. doi: 10.1054/blre.2001.0188. PMID: 11914001

Clinical prediction guides

Predictors of refractory risk in systemic lupus erythematosus-related thrombocytopenia: a dual-centre retrospective study.
Su K, Cheng H, Jia Z, Yuan Y, Yang H, Gao Q, Jiang Z, Wen H, Jiang J
Lupus Sci Med 2022 May;9(1) doi: 10.1136/lupus-2022-000677. PMID: 35606019Free PMC Article
Eltrombopag for the treatment of refractory thrombocytopenia associated with connective tissue disease.
Wang J, Dai M, Fu Q, Chen S
Sci Rep 2021 Mar 9;11(1):5459. doi: 10.1038/s41598-021-84493-2. PMID: 33750817Free PMC Article
Kaposiform lymphangiomatosis and kaposiform hemangioendothelioma: similarities and differences.
Ji Y, Chen S, Peng S, Xia C, Li L
Orphanet J Rare Dis 2019 Jul 5;14(1):165. doi: 10.1186/s13023-019-1147-9. PMID: 31277673Free PMC Article
Hunter syndrome with persistent thrombocytopenia.
Panigrahi I, Dhanorkar M, Didel S, Koganti RA
BMJ Case Rep 2019 Apr 20;12(4) doi: 10.1136/bcr-2018-226518. PMID: 31005860Free PMC Article
Efficacy and safety of tacrolimus in systemic lupus erythematosus patients with refractory thrombocytopenia: a retrospective study.
Li Y, Feng X
Lupus 2018 Jan;27(1):60-65. Epub 2017 May 31 doi: 10.1177/0961203317711011. PMID: 28566017

Recent systematic reviews

Danazol in the treatment of systemic lupus erythematosus: a qualitative systematic review.
Letchumanan P, Thumboo J
Semin Arthritis Rheum 2011 Feb;40(4):298-306. Epub 2010 Jun 11 doi: 10.1016/j.semarthrit.2010.03.005. PMID: 20541792

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