The Price of Success—The Long-Term Outcomes of Children with Craniopharyngioma—Two Institutions’ Experience

An analysis of patients below 21 years old treated due to craniopharyngioma in the years 1979–2022 was performed with the aim of evaluating the long-term outcome and treatment side-effects. The standard statistical tests were used, and 56 patients with a median age of 11 years were evaluated. Surgery was the primary treatment in 55 patients; however, in only 29 it was the only neurosurgical intervention. Eighteen children were treated with radiotherapy (RTH) in primary treatment. The most common neurosurgical side effects observed were visual and endocrine deficits and obesity, which were diagnosed in 27 (49%), 50 (91%), and 25 (52%) patients, respectively. Complications after RTH were diagnosed in 14 cases (32%). During the median follow-up of 8.4 years (range: 0.4–39.8 years), six patients died and the 5- and 10-year overall survival was 97% and 93%, respectively. Five-year progression-free survival for gross total resection, resection with adjuvant RTH, and non-radical resection alone was 83%, 68%, and 23%, respectively (p = 0.0006). Surgery combined with RTH provides comparable results to gross tumor resection in terms of oncologic outcome in craniopharyngioma patients. Adjuvant irradiation applied in primary or salvage treatment improves disease control. The rate of complications is high irrespective of improved surgical and radiotherapeutic management.

[1]  E. Kasper,et al.  An update on multimodal management of craniopharyngioma in children , 2023, Frontiers in Oncology.

[2]  T. Merchant,et al.  Proton therapy and limited surgery for paediatric and adolescent patients with craniopharyngioma (RT2CR): a single-arm, phase 2 study. , 2023, The Lancet. Oncology.

[3]  P. M. Munarriz,et al.  Multicentric and collaborative study of Spanish neurosurgical management of pediatric craniopharyngiomas: S-PedCPG.co. , 2023, Neurocirugia.

[4]  Lisa E. Mash,et al.  Comparison of neurocognitive and quality-of-life outcomes in pediatric craniopharyngioma patients treated with partial resection and radiotherapy versus gross-total resection only. , 2023, Journal of neurosurgery. Pediatrics.

[5]  L. Massimi,et al.  Clinical and oncological outcomes in single-stage versus staged surgery for pediatric craniopharyngiomas: a multicenter retrospective study , 2022, Journal of Endocrinological Investigation.

[6]  Wonhee Kim,et al.  Craniopharyngioma resection by endoscopic endonasal approach versus transcranial approach: A systematic review and meta-analysis of comparative studies , 2022, Frontiers in Oncology.

[7]  M. Chintagumpala,et al.  Response assessment in pediatric craniopharyngioma: recommendations from the Response Assessment in Pediatric Neuro-Oncology (RAPNO) working group. , 2022, Neuro-oncology.

[8]  T. Merchant,et al.  Endocrine Outcomes after Limited Surgery and Conformal Photon Radiation Therapy for Pediatric Craniopharyngioma: Long-term Results from the RT1 Protocol. , 2022, Neuro-oncology.

[9]  M. Karsy,et al.  Targeted Therapy in the Management of Modern Craniopharyngiomas. , 2022, Frontiers in bioscience.

[10]  P. Iglesias Targeted therapies in the medical management of craniopharyngioma , 2022, Pituitary.

[11]  S. Sudhanshu,et al.  Long-Term Outcomes of Paediatric-Onset Craniopharyngioma: A Retrospective Analysis from a Tertiary Care Centre in North India , 2022, Neurology India.

[12]  F. Ciardiello,et al.  Dramatic Therapeutic Response to Dabrafenib Plus Trametinib in BRAF V600E Mutated Papillary Craniopharyngiomas: A Case Report and Literature Review , 2022, Frontiers in Medicine.

[13]  D. Pasquier,et al.  Radiotherapy of benign intracranial tumours , 2021, Cancer/Radiothérapie.

[14]  T. Merchant,et al.  Radiotherapy alone for pediatric patients with craniopharyngioma , 2021, Journal of Neuro-Oncology.

[15]  C. Marras,et al.  Management of childhood-onset craniopharyngioma in Italy: A multicenter seven year follow-up study of 145 patients. , 2021, The Journal of clinical endocrinology and metabolism.

[16]  J. Sheehan,et al.  Stereotactic radiosurgery for craniopharyngiomas , 2021, Acta Neurochirurgica.

[17]  W. Lei,et al.  An analysis of clinical characteristics and postoperative complications in children craniopharyngioma , 2021, Child's Nervous System.

[18]  G. Reifenberger,et al.  The 2021 WHO Classification of Tumors of the Central Nervous System: a summary. , 2021, Neuro-oncology.

[19]  V. Mehta,et al.  Aggressive Childhood-onset Papillary Craniopharyngioma Managed With Vemurafenib, a BRAF Inhibitor , 2021, Journal of the Endocrine Society.

[20]  Jun Pan,et al.  Endoscopic endonasal versus transcranial surgery for primary resection of craniopharyngiomas based on a new QST classification system: a comparative series of 315 patients. , 2021, Journal of neurosurgery.

[21]  J. Jane,et al.  Editorial. Craniopharyngioma classification. , 2021, Journal of neurosurgery.

[22]  B. Fullerton,et al.  Proton Radiation Therapy for Pediatric Craniopharyngioma Protons for Craniopharyngioma. , 2021, International journal of radiation oncology, biology, physics.

[23]  C. Belka,et al.  ESTRO ACROP guideline for target volume delineation of skull base tumors. , 2020, Radiotherapy and oncology : journal of the European Society for Therapeutic Radiology and Oncology.

[24]  M. Chicoine,et al.  Successful Use of BRAF/MEK Inhibitors as a Neoadjuvant Approach in the Definitive Treatment of Papillary Craniopharyngioma. , 2020, Journal of the National Comprehensive Cancer Network : JNCCN.

[25]  A. Chacko,et al.  Long-term outcomes following maximal safe resection in a contemporary series of childhood craniopharyngiomas , 2020, Acta Neurochirurgica.

[26]  J. Almeida,et al.  Current Results of Surgical Treatment of Craniopharyngiomas: The Impact of Endoscopic Endonasal Approaches. , 2020, World neurosurgery.

[27]  A. Figaji,et al.  SIOP PODC–adapted treatment guidelines for craniopharyngioma in low‐ and middle‐income settings , 2020, Pediatric blood & cancer.

[28]  A. Grossman,et al.  The Medical Therapy of Craniopharyngiomas: The Way Ahead. , 2019, The Journal of clinical endocrinology and metabolism.

[29]  H. Müller The Diagnosis and Treatment of Craniopharyngioma , 2019, Neuroendocrinology.

[30]  M. Jensterle,et al.  Advances in the Management of Craniopharyngioma in Children and Adults , 2019, Radiology and oncology.

[31]  R. Carrau,et al.  Pediatric craniopharyngioma , 2019, Child's Nervous System.

[32]  R. Carrau,et al.  A modern series of subdiaphragmatic craniopharyngiomas. , 2019, Journal of neurosurgery.

[33]  M. Baine,et al.  Patterns of Care in Pediatric Craniopharyngioma: Outcomes Following Definitive Radiotherapy , 2019, AntiCancer Research.

[34]  J. Kilday,et al.  Papillary craniopharyngioma in a 4-year-old girl with BRAF V600E mutation: a case report and review of the literature , 2018, Child's Nervous System.

[35]  H. Müller Hypothalamic involvement in craniopharyngioma—Implications for surgical, radiooncological, and molecularly targeted treatment strategies , 2018, Pediatric blood & cancer.

[36]  Sean S. Park,et al.  Recurrent Papillary Craniopharyngioma with BRAF V600E Mutation Treated with Dabrafenib: A Case Report , 2018, Journal of Neurological Surgery Part B: Skull Base.

[37]  M. Link,et al.  Pediatric Craniopharyngiomas: A Primer for the Skull Base Surgeon , 2018, Journal of Neurological Surgery Part B: Skull Base.

[38]  D. Mazzatenta,et al.  Endoscopic and Microscopic Transsphenoidal Surgery of Craniopharyngiomas: A Systematic Review of Surgical Outcomes Over Two Decades , 2017, Journal of Neurological Surgery Part A: Central European Neurosurgery.

[39]  C. Catsman-Berrevoets,et al.  Very long-term sequelae of craniopharyngioma. , 2017, European journal of endocrinology.

[40]  M. Gaze,et al.  The neuroendocrine sequelae of paediatric craniopharyngioma: a 40-year meta-data analysis of 185 cases from three UK centres. , 2017, European journal of endocrinology.

[41]  A. V. van Beek,et al.  Radiotherapy, Especially at Young Age, Increases the Risk for De Novo Brain Tumors in Patients Treated for Pituitary/Sellar Lesions , 2017, The Journal of clinical endocrinology and metabolism.

[42]  D. Wheeler,et al.  Genomic Alterations of Adamantinomatous and Papillary Craniopharyngioma , 2017, Journal of neuropathology and experimental neurology.

[43]  T. Schwartz,et al.  Endoscopic endonasal versus open transcranial resection of craniopharyngiomas: a case-matched single-institution analysis. , 2016, Neurosurgical focus.

[44]  J. Eloy,et al.  Microscopic versus endoscopic approaches for craniopharyngiomas: choosing the optimal surgical corridor for maximizing extent of resection and complication avoidance using a personalized, tailored approach. , 2016, Neurosurgical focus.

[45]  Martin Sill,et al.  Adamantinomatous and papillary craniopharyngiomas are characterized by distinct epigenomic as well as mutational and transcriptomic profiles , 2016, Acta Neuropathologica Communications.

[46]  D. Louis,et al.  Dramatic Response of BRAF V600E Mutant Papillary Craniopharyngioma to Targeted Therapy. , 2016, Journal of the National Cancer Institute.

[47]  M. Eveslage,et al.  History before diagnosis in childhood craniopharyngioma: associations with initial presentation and long-term prognosis. , 2015, European journal of endocrinology.

[48]  J. Blay,et al.  Vemurafenib in Multiple Nonmelanoma Cancers with BRAF V600 Mutations. , 2015, The New England journal of medicine.

[49]  R. Beaney,et al.  Pronounced response of papillary craniopharyngioma to treatment with vemurafenib, a BRAF inhibitor , 2015, Pituitary.

[50]  S. McGovern,et al.  Proton beam therapy versus conformal photon radiation therapy for childhood craniopharyngioma: multi-institutional analysis of outcomes, cyst dynamics, and toxicity. , 2014, International journal of radiation oncology, biology, physics.

[51]  J. Debus,et al.  Long term results after fractionated stereotactic radiotherapy (FSRT) in patients with craniopharyngioma: maximal tumor control with minimal side effects , 2014, Radiation oncology.

[52]  T. Pietsch,et al.  Childhood Craniopharyngioma – Changes of Treatment Strategies in the Trials KRANIOPHARYNGEOM 2000/2007 , 2014, Klinische Pädiatrie.

[53]  John Y. K. Lee,et al.  Exome sequencing identifies BRAF mutations in papillary craniopharyngiomas , 2014, Nature Genetics.

[54]  M. Buchfelder,et al.  Surgery for craniopharyngioma , 2013, Pituitary.

[55]  K. Auguste,et al.  A systematic review of the results of surgery and radiotherapy on tumor control for pediatric craniopharyngioma , 2013, Child's Nervous System.

[56]  B. Okopień,et al.  [Diagnosis and treatment of craniopharyngioma]. , 2012, Polski merkuriusz lekarski : organ Polskiego Towarzystwa Lekarskiego.

[57]  Theodore H Schwartz,et al.  Endoscopic endonasal compared with microscopic transsphenoidal and open transcranial resection of craniopharyngiomas. , 2012, World neurosurgery.

[58]  K. Flaherty,et al.  Inhibition of mutated, activated BRAF in metastatic melanoma. , 2010, The New England journal of medicine.

[59]  Rajwant Kaur,et al.  Craniopharyngioma: a comparison of tumor control with various treatment strategies. , 2010, Neurosurgical focus.

[60]  T. Merchant,et al.  Radiation therapy for pediatric craniopharyngioma. , 2010, Neurosurgical focus.

[61]  T. Pietsch,et al.  Analyses of Treatment Variables for Patients with Childhood Craniopharyngioma – Results of the Multicenter Prospective Trial KRANIOPHARYNGEOM 2000 after Three Years of Follow-Up , 2008, Hormone Research in Paediatrics.

[62]  P. Gardner,et al.  The evolution of the endonasal approach for craniopharyngiomas. , 2008, Journal of neurosurgery.

[63]  P. Gardner,et al.  Expanded endonasal approach, a fully endoscopic transnasal approach for the resection of midline suprasellar craniopharyngiomas: a new classification based on the infundibulum. , 2008, Journal of neurosurgery.

[64]  M. Yașargil,et al.  Total removal of craniopharyngiomas. Approaches and long-term results in 144 patients. , 1990, Journal of neurosurgery.

[65]  OUP accepted manuscript , 2022, Neuro-Oncology.

[66]  M. El-Beltagy,et al.  Pediatric brain tumors in a low/middle income country: does it differ from that in developed world? , 2015, Journal of Neuro-Oncology.

[67]  S. Puget,et al.  Pediatric craniopharyngiomas: classification and treatment according to the degree of hypothalamic involvement. , 2007, Journal of neurosurgery.