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Meta-Analysis
. 2023 Nov 14;7(21):6451-6465.
doi: 10.1182/bloodadvances.2023010587.

Treatment strategy for acquired pure red cell aplasia: a systematic review and meta-analysis

Hervé Lobbes  1   2 , Jean-Christophe Lega  3   4   5 , Guillaume Le Guenno  1 , Marc Ruivard  1   2 , Sabine Mainbourg  3   5   6
Affiliations
Meta-Analysis

Treatment strategy for acquired pure red cell aplasia: a systematic review and meta-analysis

Hervé Lobbes et al. Blood Adv. .

Abstract

The treatment of autoimmune acquired pure red cell aplasia (aPRCA) is challenging. Guidelines are based on expert recommendations in the absence of controlled trials. We assessed the efficacy of the main treatment strategy through a systematic review and meta-analysis using MEDLINE, EMBASE, and the Cochrane Library up to September 2022. The overall response rate (ORR) was pooled using random-effects models. In total, 24 observational studies (19 retrospective, median follow-up of 48 months) encompassing 753 patients (49% male) were included. Primary aPRCA represented 57% of the cases. The risk of bias was moderate to high using the ROBINS-I tool. Substantial heterogeneity (I2 > 50%) was retrieved. Corticosteroids as monotherapy as first-line treatment (186 patients, 13 studies) provided an ORR of 47% (95% confidence interval [CI], 34-60). Cyclosporine A was the most frequently used immunosuppressant agent (384 patients, 18 studies), providing an ORR of 74% (95% CI, 66-82) with a similar ORR in first- (73%) and second-line (76%) treatment and when cyclosporin was used as monotherapy (83%) or with corticosteroids (77%). A total of 112 patients (10 studies) received cyclophosphamide, with an ORR of 49% (95% CI, 35-64), which was higher when cyclophosphamide was combined with corticosteroids (48%) and used in second-line treatment (58%) than in monotherapy (31%), and in first-line treatment (44%). Sirolimus use was reported only after cyclosporine A failure and provided an ORR of 87% (95% CI, 68-100; 64 patients, 3 studies). Substantial uncertainty remains regarding the best treatment strategy in the absence of high-quality evidence. This study was registered on the PROPERO database as #CRD42022360452.

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Conflict of interest statement

Conflict-of-interest disclosure: The authors declare no competing financial interests.

Figures

None
Graphical abstract
Figure 1.
Figure 1.
PRISMA flow diagram.
Figure 2.
Figure 2.
Risk of bias across domains per the ROBINS-I tool.
Figure 3.
Figure 3.
ORR per immunosuppressive strategies in the overall population (excluding thymectomy).
Figure 3.
Figure 3.
ORR per immunosuppressive strategies in the overall population (excluding thymectomy).
Figure 4.
Figure 4.
Overall response rate of the most commonly used immunosuppressive drugs in disease subgroup analysis. (A) Primary aPRCA, (B) LGLL-associated aPRCA, (C) AID-associated aPRCA, and (D) thymoma-associated aPRCA.
Figure 4.
Figure 4.
Overall response rate of the most commonly used immunosuppressive drugs in disease subgroup analysis. (A) Primary aPRCA, (B) LGLL-associated aPRCA, (C) AID-associated aPRCA, and (D) thymoma-associated aPRCA.
Figure 4.
Figure 4.
Overall response rate of the most commonly used immunosuppressive drugs in disease subgroup analysis. (A) Primary aPRCA, (B) LGLL-associated aPRCA, (C) AID-associated aPRCA, and (D) thymoma-associated aPRCA.
Figure 4.
Figure 4.
Overall response rate of the most commonly used immunosuppressive drugs in disease subgroup analysis. (A) Primary aPRCA, (B) LGLL-associated aPRCA, (C) AID-associated aPRCA, and (D) thymoma-associated aPRCA.
See this image and copyright information in PMC

References

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