Aplastic anemia in childhood is a difficult
condition to treat, and Nair and colleagues are to be commended for
their meticulous collection of single-center data over a 22-year period
[1]. They evaluated the efficacy of immunosuppressive therapy (IST) with
antithymocyte globulin (ATG) and cyclosporine (CSA) on 33 pediatric
patients with acquired aplastic anemia in the Indian setting. Their
results indicate that in India, with appropriate supportive care,
pediatric aplastic anemia patients treated with IST can achieve response
rates similar to North America and Europe, where the response rate is as
high as 81% [2]. In addition, they confirm the experience of other
authors, that ATG and CSA alone are the backbone of IST in treating
childhood aplastic anemia, and that administration of G-CSF reduced
infectious complications, but had no impact on survival or outcome [3].
Nair, et al. [1] have added valuable data on
clinical outcomes on childhood aplastic anemia in India. With an
estimated population incidence in India of less than 2 per million, such
clinical series are hard to collect and analyze. As they acknowledge,
some of their data need to be interpreted with caution: median follow-up
was only 24 months, and we know that there are significant changes in
expected survival over time. As previously emphasized, for clinical
studies in India to provide useful data, long-term follow up is of
paramount importance, and this is especially true given the known late
effects in aplastic anemia patients treated with IST [4].
The data yields other interesting trends: compared to
the 42 pediatric patients treated with IST in Toronto by Pongtanakul,
et al. [2], although response rates were similar (88 vs 81%),
there was a significant decrease in the percentage of patients with
complete response (CR) (24% vs 62%) with an increase in partial
response (PR) (64% vs 19%). This predominance of partial
responders was seen also in another Indian series reported by Chandra,
et al. [5], who raised the question of effective cyclosporine
administration, given the expense and uncertain quality in the open
market. The only other published series by George, et al. [6],
had 60 children who received ALG while 10 received ATG, with response
seen in only 43.5%, evenly divided between CR and PR. We know the nature
of the immune globulin product makes a significant difference, for
example equine-ATG has been shown to be superior to rabbit ALG in terms
of long-term response [7].
Since there is significant variation in both ATG and
CSA available in the Indian market, we can speculate whether Nair, et
al. could ensure that patients at the Army Hospital received
medications of better quality than those who had to purchase it on the
open market, but there is no easy way to verify this hypothesis. In
summary, the most important lesson we have learned is that within Indian
pediatric hematology there is no lack of knowledge or skill, and
equivalent results to any other country can be achieved. The key lies in
ensuring supply of appropriate medication and adequate supportive care,
and as the World Health Organization grapples with the control of
non-communicable disease, this is the deep-seated issue we have to
struggle with: how do we ensure that all children in India receive
access to curative healthcare?
Competing interests: None stated; Funding:
Nil
References
1. Nair V, Sondhi V, Sharma A, Das S, Sharma S.
Survival after immunosuppressive therapy in children with aplastic
anemia. Indian Pediatr. 2012;49:371-6.
2. Pongtanakul B, Das PK, Charpentier K, Dror Y.
Outcome of children with aplastic anemia treated with immunosuppressive
therapy. Pediatr Blood Cancer. 2008;50:52-7.
3. Kurre P, Johnson FL, Deeg HJ. Diagnosis and
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2005;45:770-80.
4. Arora B, Kanwar V. Childhood cancers in India:
Burden, barriers, and breakthroughs. Indian J Cancer. 2009;46:257-9.
5. Chandra J, Naithani R, Ravi R, Singh V, Narayan S,
Sharma S, et al. Antithymocyte globulin and cyclosporin in
children with acquired aplastic anemia. Indian J Pediatr. 2008;
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6. George B, Mathews V, Viswabandya A, Lakshmi KM,
Srivastava A, Chandy M. Allogeneic hematopoietic stem cell
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children with aplastic anemia—a single-center analysis of 100 patients.
Pediatr Hematol Oncol. 2010;27:122-31.
7. Scheinberg P, Nunez O, Weinstein B, Scheinberg P,
Biancotto A, Wu CO, et al. Horse versus rabbit antithymocyte
globulin in acquired aplastic anemia. N Engl J Med. 2011; 365:430-8.
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