These are reviewed studies whose abstracts concern Pineoblastoma. Each describes only what that study reported. This is not a claim by OncoForge that any compound helps or harms Pineoblastoma. Most are early lab, animal, or small human studies, and findings often conflict.
6 studies2 human⚠ Conflicting evidenceMechanism (3)Trial (1)
Tracking 6 published studies of Pineoblastoma: 2 in humans, 4 reviews/other.
Reported direction across studies: 1 positive, 2 mixed, 3 inconclusive.
Findings conflict — both supportive and negative/mixed results exist (see below). Human evidence is limited.
These counts summarize what the studies reported; they are not a measure of whether anything works for Pineoblastoma.
Human trialTrialMixed resultsModerate evidenceTier 4 · clinicaln = 77
Neuro-oncology · Oct 2025 · phase 3 randomized controlled trial
embryonal brain tumorsmedulloblastomaGroup 3 medulloblastomaSHH medulloblastomaembryonal tumor with multilayered rosettespineoblastoma
This phase 3 randomized trial tested adding high-dose methotrexate to induction chemotherapy in children ≤36 months with high-risk embryonal brain tumors. Overall complete response rates were similar between arms, but in medulloblastoma patients methotrexate was associated with higher CR (63% vs 30%) and improved 5-year event-free survival in Group 3 medulloblastoma (70% vs 33.3%). No benefit was seen for embryonal tumor with multilayered rosettes or pineoblastoma.
Reported effects: eligible patients 77, n=77 · patients evaluated for response 59, n=59 · +8 more
Studied with: induction chemotherapy, high-dose consolidation chemotherapy with hematopoietic stem-cell infusion.
Key findings
- Of 77 eligible patients, 59 with detectable disease were evaluated for response and 28 (47.5%) achieved CR; 15/30 (50%) treated with methotrexate compared to 13/29 (45%) without methotrexate (P = 0.35).
- For medulloblastoma (MB), CR was 12/19 (63%) with methotrexate compared to 6/20 (30%) without methotrexate (P = 0.039).
- All SHH subtype MB (n = 11) were survivors (molecular characterization retrospective).
- Five-year event-free survival (EFS) for Group 3 MB was 70% (90% CI: 39.6-87.2) with methotrexate versus 33.3% (90% CI: 15.0-52.9) without (P = 0.037).
- In other embryonal tumors, CR was 3/11 (27%) with methotrexate compared to 7/9 (78%) without (P = 0.99).
- No benefit observed for Embryonal Tumor with Multilayered Rosettes (n = 14; EFS 20.0% [90% CI: 1.8-52.5] with methotrexate versus 33.3% [90% CI: 10.8-58.1] without, P = 0.58) or pineoblastoma (n = 9; EFS 16.7% [90% CI: 1.6-46.1] with methotrexate versus 0% without, P = 0.52).
Limitations: Relatively small overall sample size (77 eligible) with smaller numbers in histologic/molecular subgroups; Molecular characterization was conducted retrospectively; Some subgroup analyses involve very small n (e.g., Group 3 MB: 10 vs 15; SHH MB n=11); Tests of significance were one-sided (as stated); Confidence intervals reported are 90% rather than the more conventional 95%.
AI summary of the abstract, human-reviewed · Jun 2026. Describes what this study reported, not medical advice. View on PubMed · Full text
ReviewMixed resultsLimited evidenceTier 4 · clinical
Neuro-oncology · Dec 2024 · consensus review
pineal parenchymal tumorspineocytomapineal parenchymal tumor of intermediate differentiationpineoblastomapapillary tumor of the pineal region
This international consensus review summarizes diagnostic and treatment approaches for rare pineal parenchymal tumors and related intrinsic pineal masses. It highlights recent genomic findings that informed refinements in the WHO 5th edition molecular classification and offers pragmatic clinical management recommendations ranging from surgery alone to intensive multimodal antineoplastic therapy.
Key findings
- Pineal parenchymal tumors are rare and lack robust evidence-based treatment recommendations.
- These tumors vary in biology, clinical characteristics, and prognosis, necessitating a range of treatments from surgical resection alone to intensive multimodal antineoplastic therapy.
- Recent international genomic studies have refined the molecular-based disease classification, incorporated in the WHO 5th edition.
- The review summarizes literature on diagnostic and therapeutic approaches and suggests pragmatic recommendations for clinical management of intrinsic pineal region masses (pineocytoma, PPTID, pineoblastoma), pineal cyst, and papillary tumors of the pineal region.
Limitations: Tumors are rare and high-quality evidence is sparse, limiting strength of recommendations.; Recommendations are based on literature review and consensus rather than randomized controlled trials.; Heterogeneity of tumor biology and prognosis may limit generalizability of suggested management approaches.; No new primary quantitative data are reported in this review..
AI summary of the abstract, human-reviewed · Jun 2026. Describes what this study reported, not medical advice. View on PubMed · Full text
ReviewMechanismInconclusiveLimited evidenceTier 3 · early human
Acta neuropathologica · Apr 2020
pleuropulmonary blastomaovarian Sertoli-Leydig cell tumorcystic nephromamultinodular goiterthyroid carcinomaanaplastic sarcoma of the kidneyembryonal rhabdomyosarcomanasal chondromesenchymal hamartomametastases of pleuropulmonary blastoma to the cerebrumpituitary blastomapineoblastomaciliary body medulloepitheliomaprimary DICER1-associated CNS sarcomasETMR-like infantile cerebellar embryonal tumormacrocephaly (non-neoplastic phenotype)
This is a review of the central nervous system (CNS) manifestations of DICER1 syndrome, a rare tumor predisposition syndrome that mainly affects children and young adults. The authors summarize the genetic basis (germline loss-of-function DICER1 alterations with somatic RNase IIIb hotspot missense mutations), the wide spectrum of pleiotropic benign and malignant lesions with pleuropulmonary blastoma as the hallmark tumor, and previously defined CNS manifestations including several primary CNS tumors and macrocephaly as a non-neoplastic phenotype.
Key findings
- DICER1 syndrome is typically caused by heterozygous germline loss-of-function DICER1 alterations accompanied by somatic missense mutations at hotspots in the RNase IIIb domain.
- DICER1 encodes a component of the microRNA biogenesis machinery.
- The syndrome is highly pleiotropic and includes a constellation of benign and malignant neoplastic and dysplastic lesions.
- Pleuropulmonary blastoma (PPB) is the hallmark tumor of the syndrome.
- Other reported manifestations include ovarian Sertoli-Leydig cell tumor, cystic nephroma arising in childhood, multinodular goiter, thyroid carcinoma, anaplastic sarcoma of the kidney, embryonal rhabdomyosarcoma, and nasal chondromesenchymal hamartoma.
- CNS manifestations defined in the literature include PPB metastases to the cerebrum, pituitary blastoma, pineoblastoma, ciliary body medulloepithelioma, primary DICER1-associated CNS sarcomas, and ETMR-like infantile cerebellar embryonal tumor.
- Macrocephaly has been reported as a non-neoplastic, haploinsufficient phenotype associated with DICER1.
Limitations: Review article: no new primary patient-level data presented in the abstract.; DICER1 syndrome is rare, so the literature is likely composed of small case series and individual reports, limiting generalizability.; Abstract does not indicate systematic review methods or quantitative synthesis.; Heterogeneous and pleiotropic manifestations make it difficult to derive uniform clinical conclusions from a narrative review..
AI summary of the abstract, human-reviewed · Jun 2026. Describes what this study reported, not medical advice. View on PubMed
ReviewMechanismInconclusiveModerate evidenceTier 3 · early human
Klinicka onkologie : casopis Ceske a Slovenske onkologicke spolecnosti · Jul 2019
pleuropulmonary blastomamultinodular goiterovarian Sertoli-Leydig cell tumorcystic nephromamedulloepithelioma (ciliary body/iris)embryonal rhabdomyosarcoma (botryoid type)nasal epithelial/chondromesenchymal hamartomapituitary blastomapineoblastomadifferentiated thyroid carcinomapulmonary blastomawell-differentiated fetal lung adenocarcinomaanaplastic sarcoma of the kidneyprimary ovarian sarcomaPPB-like peritoneal sarcomamulticystic liver neoplasmsWilms tumor
This article summarizes the clinical features, genetic diagnosis, management, and surveillance recommendations for DICER1 syndrome, an inherited disorder caused by germline DICER1 pathogenic variants that predispose to a spectrum of benign and malignant tumors. It lists the most common associated tumors (e.g., pleuropulmonary blastoma, thyroid nodules, ovarian Sertoli-Leydig cell tumors) and gives recommended surveillance schedules and guidance on genetic testing and cascade testing for relatives. The authors note autosomal dominant inheritance with reduced penetrance and state diagnosis is by identification of a heterozygous germline DICER1 pathogenic variant.
Key findings
- DICER1 syndrome is caused by pathogenic variants in the DICER1 gene (located at chromosome 14q32.13) and is associated with increased risk of a spectrum of malignant and benign tumors.
- The most common clinical features include lung cysts and thyroid nodules; common neoplasms include pleuropulmonary blastoma, Sertoli-Leydig cell tumor, pediatric cystic nephroma, and differentiated thyroid carcinoma.
- A broad and variable tumor spectrum is reported, with many tumors occurring before age 40 and PPB typically presenting in children younger than seven years.
- Diagnosis is established by identification of a heterozygous germline DICER1 pathogenic variant presumed to cause loss of function.
- Management of DICER1-associated tumors is tumor-type dependent and may include surgery, chemotherapy, and in some cases radiation; surveillance recommendations (based on the 2016 International PPB Register) are provided for chest imaging, thyroid ultrasound, pelvic and abdominal ultrasound, ophthalmologic assessment, and neurologic monitoring.
- Genetic counseling is recommended, with cascade testing of at-risk first-degree relatives and consideration of testing soon after birth because screening often begins in infancy.
Limitations: This is a review/clinical overview rather than original primary quantitative research.; Surveillance recommendations are presented but the abstract does not provide quantitative evidence of their effectiveness.; Recommendations appear to be based on existing guidance (2016 International PPB Register) and may reflect expert consensus rather than prospective trial data.; Variable penetrance and broad tumor spectrum limit precise risk prediction for individual carriers..
AI summary of the abstract, human-reviewed · Jun 2026. Describes what this study reported, not medical advice. View on PubMed
ReviewInconclusiveLimited evidenceTier 3 · early human
Clinical cancer research : an official journal of the American Association for Cancer Research · May 2018
pleuropulmonary blastoma (PPB)ovarian sex cord-stromal tumorsSertoli-Leydig cell tumorlung cystscystic nephromarenal sarcomaWilms tumornodular hyperplasia of the thyroidnasal chondromesenchymal hamartomaciliary body medulloepitheliomagenitourinary embryonal rhabdomyosarcomapineoblastomapituitary blastomagastrointestinal polyps
This paper reports an expert consensus from the inaugural International DICER1 Symposium summarizing genetic testing and surveillance recommendations for people with pathogenic germline DICER1 variants. It lists the spectrum of associated tumors and lesions, recommends education and imaging-based surveillance (with emphasis that risks are highest in early childhood and decline in adulthood), and states that guidelines will be updated as research expands.
Key findings
- Pathogenic germline DICER1 variants cause a hereditary cancer predisposition syndrome with a variety of manifestations.
- Associated neoplasms and lesions include pleuropulmonary blastoma (PPB), ovarian sex cord-stromal tumors (particularly Sertoli-Leydig cell tumor), lung cysts, cystic nephroma, renal sarcoma and Wilms tumor, nodular hyperplasia of the thyroid, nasal chondromesenchymal hamartoma, ciliary body medulloepithelioma, genitourinary embryonal rhabdomyosarcoma, and brain tumors including pineoblastoma and pituitary blastoma.
- The International PPB Registry convened a multidisciplinary international symposium to develop consensus testing, surveillance, and treatment recommendations.
- Recommendations are provided for genetic testing, prenatal management, and surveillance for DICER1-associated pulmonary, renal, gynecologic, thyroid, ophthalmologic, otolaryngologic, and central nervous system tumors and gastrointestinal polyps.
- Risk for most DICER1-associated neoplasms is highest in early childhood and decreases in adulthood.
- Individual and caregiver education and judicious imaging-based surveillance are the primary recommended approaches.
- These recommendations reflect a consensus of expert opinion and current literature and will be updated as DICER1 research expands.
Limitations: Recommendations are based on expert consensus and existing literature rather than new primary data reported in this article.; Abstract does not present quantitative risk estimates, systematic evidence grading, or methodological details of literature review.; Guidance may change as further research in DICER1-associated conditions emerges..
AI summary of the abstract, human-reviewed · Jun 2026. Describes what this study reported, not medical advice. View on PubMed · Full text
Human · observationalMechanismReported positiveLimited evidenceTier 3 · early humann = 16
Pediatric radiology · Sep 2017 · retrospective analysis
pleuropulmonary blastomapineoblastomaovarian Sertoli-Leydig cell tumorembryonal rhabdomyosarcomarenal sarcomacystic nephromathyroid nodule/cystrenal cystpineal cyst
This retrospective review analyzed imaging from 16 pediatric patients (≤18 years) with germline DICER1 variants seen from January 2004 to July 2016. The authors report a spectrum of DICER1-associated malignant tumors (in 68.8% of patients) and benign lesions (in 37.5%), and describe a common imaging appearance they call the "cracked windshield" sign. They conclude that early surveillance of at-risk patients is important while minimizing ionizing radiation exposure.
Reported effects: patients included 16, n=16 · female patients 12, n=16 · +13 more
Key findings
- Sixteen patients were included (12 females; mean age at presentation: 4.2years, range: 14days to 17years), with surveillance imaging encompassing chest X-ray and CT; abdominal, pelvic and neck US; and brain and whole-body MRI.
- Malignant lesions (68.8% of patients) included pleuropulmonary blastoma (5), pineoblastoma (3), ovarian Sertoli-Leydig cell tumor (1), embryonal rhabdomyosarcoma (1) and renal sarcoma (1).
- Benign lesions (37.5% of patients) included thyroid cysts (2), thyroid nodules (2), cystic nephroma (2), renal cysts (1) and pineal cyst (1).
- A common lesional appearance observed across modalities and organs was defined as the "cracked windshield" sign.
- Authors suggest early surveillance of at-risk patients while minimizing exposure to ionizing radiation.
Limitations: Retrospective design; Small sample size (n=16); Single tertiary pediatric centre (potential limited generalizability); Descriptive imaging study without control group or assessment of outcomes of surveillance; Possible selection bias from keyword-driven imaging database search and cross-referencing with institutional genetics database.
AI summary of the abstract, human-reviewed · Jun 2026. Describes what this study reported, not medical advice. View on PubMed