Curr Med SCI: a novel immunosuppressive therapy for children with very severe aplastic anemia with autoantibodies

Currently, many patients with very severe aplastic anemia (VSAA) are unable to receive hematopoietic stem cell transplantation (HSCT) or standard immunosuppressive therapy (IST) due to the high cost of treatment, and there is a shortage of sibling donors and a lack of resources
to support HSCT.
In addition, some patients with VSAA with autoantibodies do not have a life-threatening infection or bleeding
at the time of initial diagnosis.
Taking into account disease status, economics and other factors, this study devised a new relatively mild treatment strategy: cyclosporine A plus pulsed high-dose prednisone (CsA HDP).

Figure 1: Overall survival of IST (CsA+HDP) patients with very severe aplastic anemia

Figure 2: IST (CsA+HDP) survival without failure in patients with very severe aplastic anemia

The study retrospectively analyzed 11 VSAA patients
who received CsA + HDP treatment in our hospital from August 2017 to August 2019.
The median follow-up of these patients was 24
.
9 months
.
The overall response rate was 54
.
5% (6/11) six months after IST launch and 81
.
The deadline is 8% (9/11).

At the last follow-up, five patients achieved a complete response and four patients met criteria for
a partial response.
The median response time for respondents was 110 days
.
Three patients received HSCT
due to poor CsA HDP or the search for a suitable transplant donor.
Relapse and clonal evolution
were not found in these patients.
The estimated 3-year overall survival and 3-year failure-free survival rate is 100
.
0% and 72
.
7%
respectively.
In addition, the results showed that cyclosporine-prednisone-related toxicity was mild and well
tolerated in most patients.

Table 1: Prognosis and adverse reactions of patients

In general, whether it is HSCT, standard IST or other treatments, the treatment of AA and VSAA in particular is a long-term, complex and depleting process
.
In clinical practice, patients sometimes abandon treatment due to financial difficulties, resulting in a poor
prognosis.
Given the low efficacy, safety, and adverse effects, the CsA+HDP regimen may provide a new option
for patients who cannot receive MFDHSCT and standard IST in a timely manner.

Original source:

Wang ZJ, Chen HB, Zhou F, Yu H, Wu XY, Shen YQ, Qiu YN, Jin RM.
A New Immunosuppressive Therapy for Very Severe Aplastic Anemia in Children with Autoantibodies.
Curr Med Sci.
2022 Apr; 42(2):379-386.
doi: 10.
1007/s11596-022-2519-2.
Epub 2022 Mar 8.
PMID: 35258748.