[evansmom]

Hi Trish,

I trust Jessica's specialist is not suggesting another round of ATG yet. If I'm not mistaken, it's only been about a month. Most responses take 3-6 months to happen. There are studies that have taken numerous cases of pediatric AA treatment courses and their outcomes and this is where the recommendation to go to transplant comes from, if there is a suitable donor:

Prospective multicenter trial comparing repeated immunosuppressive therapy with stem-cell transplantation from an alternative donor as second-line treatment for children with severe and very severe aplastic anemia.

Kosaka, Yoshiyuki. Yagasaki, Hiroshi. Sano, Kimihiko. Kobayashi, Ryoji. Ayukawa, Hiroshi. Kaneko, Takashi. Yabe, Hiromasa. Tsuchida, Masahiro. Mugishima, Hideo. Ohara, Akira. Morimoto, Akira. Otsuka, Yo****oshi. Ohga, Shouichi. Bessho, Fumio. Nakahata, Tatsutoshi. Tsukimoto, Ichiro. Kojima, Seiji. Japan Childhood Aplastic Anemia Study Group.


Blood. 111(3):1054-9, 2008 Feb 1


We conducted a prospective multicenter study to compare the efficacy of repeated immunosuppressive therapy (IST) with stem-cell transplantation (SCT) from an alternative donor in children with acquired aplastic anemia (AA) who failed to respond to an initial course of IST.

Patients with severe (n = 86) and very severe disease (n = 119) received initial IST consisting of antithymocyte globulin (ATG) and cyclosporine. Sixty patients failed to respond to IST after 6 months from the initial IST and were eligible for second-line treatment.

Among them, 21 patients lacking suitable donors received a second course of IST. Three patients developed an anaphylactoid reaction to ATG and could not complete the second IST.

A trilineage response was seen in only 2 of 18 (11%) evaluable patients after 6 months.

Thirty-one patients received SCT from an alternative donor.

At 5 years from the initiation of second-line therapy, the estimated failure-free survival (FFS), defined as survival with response, was 83.9% (+/- 16.1%, SD) in the SCT group compared with 9.5% (+/- 9.0%) in the IST group (P = .001).

These results suggest that SCT from an alternative donor offers a better chance of FFS than a second IST in patients not responding to an initial IST.

Although AA is unlike leukemias with their very specific tried and true protocols depending on the type, there are still basic protocols that have been developed based on research and studies to treat AA and there are differences on the routes best taken depending on the age of the aplastic patient. Please make sure the doctor treating Jessica has familiarized himself with the pediatric treatment course for best possible outcome.

Arm yourself with as much information as possible, you have involuntarily enrolled in a Bone Marrow 101 rapid study course.

Praying for Jessica's return of good health ASAP,