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. 2020 Jun;67(6):e28252.
doi: 10.1002/pbc.28252. Epub 2020 Mar 18.

Pineoblastoma in children less than six years of age: The Head Start I, II, and III experience

Mohamed S Abdelbaki  1 , Mohammad H Abu-Arja  2 , Tom B Davidson  3 , Jason R Fangusaro  4 , Joseph R Stanek  1 , Ira J Dunkel  5 , Girish Dhall  6 , Sharon L Gardner  7 , Jonathan L Finlay  1
Affiliations

Pineoblastoma in children less than six years of age: The Head Start I, II, and III experience

Mohamed S Abdelbaki et al. Pediatr Blood Cancer. 2020 Jun.

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.

Keywords: Head Start; autologous hematopoietic cell rescue; craniospinal irradiation; marrow-ablative chemotherapy; pineoblastoma.

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Conflict of interest statement

Conflict of Interest: Ira J. Dunkel is a consultant for Apexigen, Bayer, and Celgene (all unpaid or de minimis compensation). All other authors have no conflicts of interests to disclose.

Figures

Figure 1
Figure 1
Flow diagram of the 23 patients with pineoblastoma enrolled in the HS I-III trials.
Figure 2
Figure 2
Progression-free survival and overall survival for the 23 patients with pineoblastoma enrolled in the Head Start I-III trials.
Figure 3
Figure 3
Progression-free and overall survival of the 23 patients with pineoblastoma enrolled in the Head Start I-III (A,B) stratified by craniospinal irradiation and (C,D) stratified by marrow-ablative chemotherapy followed by autologous hematopoietic cell rescue.
See this image and copyright information in PMC

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