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Re‐thinking transplant for neuroblastoma

B. Kushner

Published 2021 in Pediatric Blood & Cancer

ABSTRACT

In this issue of Pediatric Blood & Cancer, Yamazaki et al. present results with myeloablative therapy (MAT) using thiotepa-melphalan in patients with high-risk neuroblastoma (HR-NB) at three institutions.1 Amodification of the regimenwas recently approved in Japan, suggesting itmight be adopted nationally. The report covers 41 patientswhose other treatments variedwidely. Thus, there was no standard for induction chemotherapy, radiotherapy, timing of tumor resection, use of 13cis-retinoic acid, or post-transplant therapies. Biostatistical analyses mitigated drawbacks of this heterogeneity. In a multivariate analysis, good response to induction proved prognostic, with post-transplant 5year event-free survival (EFS) at 50% in patients who underwent MAT in partial remission or better. This outcome is similar to experience elsewhere with MAT using melphalan plus other agents and limited post-transplant immunotherapy, for example, 3-year EFS of 38–50%.2 These figures of course are far higher than EFS of all newly diagnosed patients some of whom do not become candidates for MAT. Indeed, 15/41 (37%) patients had abnormal renal function at enrollmentwhich prompted reduced dosing of thiotepa andmelphalan and suggests that other patients likelywere excluded not only because of poor responses to induction, but because of excessive toxicity of major organs or toxic death during induction. The authors accept as a given that MAT is needed for cure of HRNB. Yet, ongoing therapeutic advances support continually reviewing data with the goal of identifying changes in treatment that promise to improve cure rates and reduce toxicity. Re-thinking MAT for HRNB is warranted. Once considered an exciting treatment for breast cancer and other solid tumors, MAT proved disappointing as experience showed exorbitant toxicity without survival benefit (see Pulitzer Prize best seller S. Mukherjee’s Emperor of All Maladies). MAT was discontinued for all extra-cranial pediatric solid tumors, aside from HRNB for which it became standard because of favorable results in the only randomized NB studies (n = 3) comparing transplant versus no transplant.3–5

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