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Pineoblastoma in children less than six years of age: The Head Start I, II, and III experience
Author(s) -
Abdelbaki Mohamed S.,
AbuArja Mohammad H.,
Davidson Tom B.,
Fangusaro Jason R.,
Stanek Joseph R.,
Dunkel Ira J.,
Dhall Girish,
Gardner Sharon L.,
Finlay Jonathan L.
Publication year - 2020
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.28252
Subject(s) - medicine , hazard ratio , chemotherapy , induction chemotherapy , confidence interval , surgery
Abstract Background We report the outcomes of patients with pineoblastoma and trilateral retinoblastoma syndrome enrolled on the Head Start (HS) I‐III trials. Methods Twenty‐three children were enrolled prospectively between 1991 and 2009. Treatment included maximal surgical resection followed by five cycles of intensive chemotherapy and consolidation with marrow‐ablative chemotherapy and autologous hematopoietic cell rescue (HDCx/AuHCR). Irradiation following consolidation was reserved for children over six years of age or those with residual tumor at the end of induction. Results Median age was 3.12 years (range, 0.44‐5.72). Three patients withdrew from the study treatment and two patients experienced chemotherapy‐related death. Eight patients experienced progressive disease (PD) during induction chemotherapy and did not proceed to HDCx/AuHCR. Ten patients received HDCx/AuHCR; eight experienced PD post‐consolidation. Seven patients received craniospinal irradiation (CSI) with a median dose of 20.7 Gy (range, 18‐36 Gy) with boost(s) (median dose 27 Gy; range, 18‐36 Gy); three received CSI as adjuvant therapy (two post‐HDCx/AuHCR) and four upon progression/recurrence. The five‐year progression‐free survival (PFS) and overall survival (OS) were 9.7% (95% confidence intervals [CI]: 2.6%‐36.0%) and 13% (95% CI: 4.5%‐37.5%), respectively. Only three patients survived beyond five years. Favorable OS prognostic factors were CSI (hazard ratio [HR] = 0.30 [0.11‐0.86], P = 0.025) and HDCx/AuHCR (HR = 0.40 [0.16‐0.99], P = 0.047). Conclusions Within the HS I‐III trials, CSI and HDCx/AuHCR were statistically associated with improved survival. The high PD rate during later induction cycles and following consolidation chemotherapy warrants consideration of fewer induction cycles prior to consolidation and the potential intensification of consolidation with multiple cycles of marrow‐ablative chemotherapy and irradiation.