Long-term follow-up of high-risk neuroblastoma survivors treated with high-dose chemotherapy and stem cell transplantation rescue

Sandrine Haghiri, Chiraz Fayech, Imène Mansouri, Christelle Dufour, Claudia Pasqualini, Stéphanie Bolle, Sophie Rivollet, Agnès Dumas, Amel Boumaraf, Amel Belhout, Neige Journy, Vincent Souchard, Giao Vu-Bezin, Cristina Veres, Nadia Haddy, Florent De Vathaire, Dominique Valteau-Couanet, Brice Fresneau

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    Abstract

    Intensive treatments including high-dose chemotherapy (HDC) with autologous stem cell rescue have improved high-risk neuroblastoma (HRNB) survival. We report the long-term health status of 145 HRNB survivors, alive and disease-free 5 years post HDC. Median follow-up was 15 years (range = 5–34). Six patients experienced late relapses, 11 developed second malignant neoplasms (SMNs), and 9 died. Event-free and overall survivals 20 years post HDC were 82% (95% CI = 70%–90%) and 89% (78%–95%), respectively. Compared with the French general population, the standardized mortality ratio was 19 (95% CI = 8.7–36.1; p < 0.0001) and the absolute excess risk was 37.6 (19.2–73.5). Late effects were observed in 135/145 patients (median = 3 events/patient); 103 had at least one severe event. SMNs arose at a median of 20 years post HDC and included carcinoma (n = 5), sarcoma (2), acute myeloid leukemia (2), melanoma (1), and malignant glioma (1). Non-oncologic health events included dental maldevelopment (60%), severe hearing loss (20% cumulative probability at 15 years), hepatic focal nodular hyperplasia (14%), thyroid (11%), cardiac (8%), and renal (7%) diseases and growth retardation (height-for-age z-score ≤ −2 for 21%). Gonadal insufficiency was near-universal after busulfan (40/43 females, 33/35 males). Severe late effects are frequent and progressive in HRNB survivors needing systematic very long-term follow-up.

    Original languageEnglish
    Pages (from-to)1984-1997
    Number of pages14
    JournalBone Marrow Transplantation
    Volume56
    Issue number8
    DOIs
    Publication statusPublished - 1 Aug 2021

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