Choroid plexus carcinoma in children: the Head Start experience

Area of Science:

• Pediatric Oncology
• Neuro-oncology
• Chemotherapy

Background:

• Choroid plexus carcinoma (CPC) is a rare, aggressive brain tumor primarily affecting young children.
• Optimal therapeutic strategies for CPC remain undefined, with a historically poor prognosis.
• Head Start (HS) regimens aim to improve survival and minimize neurocognitive deficits in young children with brain tumors by avoiding irradiation.

Purpose of the Study:

• To evaluate the efficacy of Head Start (HS) chemotherapy regimens in treating newly diagnosed choroid plexus carcinoma (CPC) in young children.
• To assess the feasibility of an irradiation-avoiding strategy in this patient population.

Main Methods:

• Three sequential Head Start (HS) studies were conducted between 1991 and 2009.
• Treatment involved maximal surgical resection followed by intensive induction chemotherapy, myeloablative chemotherapy with autologous hematopoietic stem cell rescue (AuHCR).
• Cranial irradiation was reserved for patients with residual disease or tumor progression/recurrence.

Main Results:

• Twelve children with CPC (median age 19.5 months) were treated with HS regimens.
• Five patients remain alive and disease-free without irradiation at 29-89 months post-diagnosis.
• The 3- and 5-year progression-free survival rates were 58% and 38%, respectively; overall survival rates were 83% and 62%.

Conclusions:

• Head Start (HS) chemotherapy strategies may achieve long-term remission in young children with newly diagnosed CPC.
• These regimens offer a potential alternative to cranial irradiation, minimizing associated neurocognitive sequelae.