SARS‐CoV‐2 infection coincident with newly diagnosed severe aplastic anemia: A report of two cases

Abstract We performed a prospective randomized trial of antithoracic duct lymphocyte globulin (ATDLG), HLA-haploidentical marrow, and androgen (regimen ABA) versus androgen alone (concurrent STANDARD care controls) in 42 newly diagnosed individuals with severe aplastic anemia. ABA patients also were matched with patients from our preceding study (historical STANDARD care controls). Supportive care and pretreatment patient characteristics were the same in all groups. By life table analysis, 76% of patients receiving ABA are alive at 2 yr compared to 31% of the concurrent control group (p less than 0.002 versus ABA) and 19% of the historical controls (p less than 0.0001 versus ABA) given STANDARD care. ABA patients had greater hematologic improvement than either control group (p less than 0.001). However, improvement with ABA was often incomplete. Toxicity of ATDLG was considerable but manageable. Further studies to determine the mechanism of action and active component(s) of ABA are indicated.

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Antithoracic duct lymphocyte globulin therapy of severe aplastic anemia

Blood ◽

10.1182/blood.v62.4.883.bloodjournal624883 ◽

1983 ◽

Vol 62 (4) ◽

pp. 883-888 ◽

Cited By ~ 1

Author(s):

B Camitta ◽

RJ O'Reilly ◽

L Sensenbrenner ◽

J Rappeport ◽

R Champlin ◽

...

Keyword(s):

Aplastic Anemia ◽

Standard Care ◽

Patient Characteristics ◽

Severe Aplastic Anemia ◽

Control Group ◽

Newly Diagnosed ◽

Concurrent Control ◽

Table Analysis ◽

Historical Controls ◽

Concurrent Control Group

We performed a prospective randomized trial of antithoracic duct lymphocyte globulin (ATDLG), HLA-haploidentical marrow, and androgen (regimen ABA) versus androgen alone (concurrent STANDARD care controls) in 42 newly diagnosed individuals with severe aplastic anemia. ABA patients also were matched with patients from our preceding study (historical STANDARD care controls). Supportive care and pretreatment patient characteristics were the same in all groups. By life table analysis, 76% of patients receiving ABA are alive at 2 yr compared to 31% of the concurrent control group (p less than 0.002 versus ABA) and 19% of the historical controls (p less than 0.0001 versus ABA) given STANDARD care. ABA patients had greater hematologic improvement than either control group (p less than 0.001). However, improvement with ABA was often incomplete. Toxicity of ATDLG was considerable but manageable. Further studies to determine the mechanism of action and active component(s) of ABA are indicated.

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A randomized controlled study in patients with newly diagnosed severe aplastic anemia receiving antithymocyte globulin (ATG), cyclosporine, with or without G-CSF: a study of the SAA Working Party of the European Group for Blood and Marrow Transplantation

Blood ◽

10.1182/blood-2010-08-304071 ◽

2011 ◽

Vol 117 (17) ◽

pp. 4434-4441 ◽

Cited By ~ 124

Author(s):

André Tichelli ◽

Hubert Schrezenmeier ◽

Gérard Socié ◽

Judith Marsh ◽

Andrea Bacigalupo ◽

...

Keyword(s):

Aplastic Anemia ◽

Antithymocyte Globulin ◽

Randomized Study ◽

Working Party ◽

Severe Aplastic Anemia ◽

Controlled Study ◽

Newly Diagnosed ◽

Entire Cohort ◽

Post Hoc ◽

To Receive

Abstract We evaluated the role of granulocyte colony-stimulating factor (G-CSF) in patients with severe aplastic anemia (SAA) treated with antithymocyte globulin (ATG) and cyclosporine (CSA). Between January 2002 and July 2008, 192 patients with newly diagnosed SAA not eligible for transplantation were entered into this multicenter, randomized study to receive ATG/CSA with or without G-CSF. Overall survival (OS) at 6 years was 76% ± 4%, and event-free survival (EFS) was 42% ± 4%. No difference in OS/EFS was seen between patients randomly assigned to receive or not to receive G-CSF, neither for the entire cohort nor in subgroups stratified by age and disease severity. Patients treated with G-CSF had fewer infectious episodes (24%) and hospitalization days (82%) compared with patients without G-CSF (36%; P = .006; 87%; P = .0003). In a post hoc analysis of patients receiving G-CSF, the lack of a neutrophil response by day 30 was associated with significantly lower response rate (56% vs 81%; P = .048) and survival (65% vs 87%; P = .031). G-CSF added to standard ATG and CSA reduces the rate of early infectious episodes and days of hospitalization in very SAA patients and might allow early identification of nonresponders but has no effect on OS, EFS, remission, relapse rates, and mortality. This study was registered at www.clinicaltrials.gov as NCT01163942.

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Outcome of Standard Immunosuppressive Therapy with or without Eltrombopag for Chinese Patients with Severe Aplastic Anemia in the Real World

Blood ◽

10.1182/blood-2019-131670 ◽

2019 ◽

Vol 134 (Supplement_1) ◽

pp. 5019-5019

Author(s):

Jinsong JIA ◽

Hao Jiang ◽

Xiao Jun Huang

Keyword(s):

Aplastic Anemia ◽

Immunosuppressive Therapy ◽

Real World ◽

Antithymocyte Globulin ◽

Clonal Evolution ◽

Chinese Patients ◽

Severe Aplastic Anemia ◽

Newly Diagnosed ◽

The Real ◽

Overall Response

BACKGROUND Immunosuppression is the current standard therapy for patients with severe aplastic anemia(SAA), whereas allogeneic haematopoietic stem cell transplantation (HSCT) is recommended for patients younger than 50 years old, if donors are available. Immunosuppression with antithymocyte globulin (ATG) and ciclosporin results in an overall response rate (ORR) of about 70%. In Chinese SAA patients, porcine ATG and rabbit ATG resulted apparently in similar ORRs and survivals. The low CR rate of immunosuppression with ATG suggests that other strategies are needed to improve outcome. Eltrombopag is an oral thrombopoietin receptor agonist. In a recent study of newly diagnosed SAA patients, combined standard immunosuppressive therapy (IST) with eltrombopag, the patients in one of three cohorts who received eltrombopag from day 1 to 6 months achieved higher ORR at 6 months (94%) and CR rate(58%). The results showed a higher rate of treatment response than in the past. But the data of eltrombopag in SAA have so far been limited to clinical trials, and its efficacy in routine practice remains undefined, especially for Chinese SAA patients. Recently, we retrospectively analyzed the results of 48 cases suffer from SAA received standard immunotherapy combined with or without eltrombopag in our center, so as to explore the effects and adverse reactions in the real world. METHODS 48 consecutive newly diagnosed patients with severe aplastic anemia were enrolled in this study. Out of 48 cases, 27 were male, and 21 were female. The median age was 46 years old (ranged from 18 to 69 years old). SAA patients who received ATG combined with cyclosporine A (CsA) therapy with or without eltrombopag were analysed retrospectively. The patients were divided into a eltrombopag group (n = 14) and a without eltrombopag group (n = 34). The eltrombopag group received eltrombopag (median daily dose 75 mg) from the beginning of IST and for at least 3 months. The primary outcome was complete hematologic response at 6 months. At the same time, overall response, survival, relapse, and clonal evolution to myeloid cancer were observed. RESULTS After three months, the eltrombopag group and without eltrombopag group had an ORR of 69.5% and 47.2%, respectively (P = 0.063); after six months, the ORR reached 85.7% and 73.6%, respectively (P = 0.078). The rates of complete response at 6 months was 35.7% in the eltrombopag group and 29.4% in without eltrombopag group. The complete and overall response rates in the eltrombopag group were higher than in without eltrombopag group. However, due to the small number of cases, there was no statistical difference. At a median follow-up of 2 years, the survival rate was 97.9%; one patient died from a hematologic cause. Rates of relapse and clonal evolution were similar in two gruops. Adverse effects included reversible rashes, dyspepsia, and liver function derangement. CONCLUSIONS Rates of hematologic response among chinese patients with severe aplastic anemia treated with standard immunosuppressive therapy plus eltrombopag was markedly higher than without eltrombopag. 【Key words】 Aplastic anaemia; newly diagnosed; eltrombopag; antithymocyte globulin; Disclosures No relevant conflicts of interest to declare.

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