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The Current Treatment of Childhood Cancer
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The Current Treatment of Childhood Cancer

A special issue of Journal of Clinical Medicine (ISSN 2077-0383). This special issue belongs to the section "Oncology".

Deadline for manuscript submissions: closed (31 December 2021) | Viewed by 33967

Special Issue Editor

Dr. Raushan Kurmasheva

E-Mail Website
Guest Editor
Greehey Children’s Cancer Research Institute, University of Texas Health Science Center at San Antonio, San Antonio, TX 78229, USA
Interests: pediatric cancer; molecular target; nanomedicine; precision therapy
Special Issues, Collections and Topics in MDPI journals

Special Issue Information

Dear Colleagues,

In the last four decades, the 5-year survival rate of children with cancer has increased from 58% to 83%, with the standard of care for these children relying on surgery, radiation therapy, and systemic therapy using cytotoxic agents. While cytotoxic therapies may induce complete responses in children with metastatic or relapsed tumors, however, treatment is rarely curative, and the long-term consequences of chemoradiation can be devastating. Many pediatric sarcomas are driven by fusion oncogenes resulting from chromosomal translocations (Ewing sarcoma, osteosarcoma, rhabdomyosarcoma) or by copy number changes (neuroblastoma), and recent large-scale sequencing studies (TARGET, pediatric preclinical testing consortium (PPTC), etc.) have identified frequent somatic mutations in sarcoma, neuroblastoma, and brain tumors aiding development of novel targeted therapies. Innovative approaches in pediatric oncology are aimed at enhancing the activity of chemotherapeutic agents through modulation of DNA damage response pathways. For example, the use of PARP inhibitors that exploit deficiency in homologous recombination in BRCA-deficient cancers has confirmed the concept of “synthetic lethality”. In pediatric preclinical testing, PARP inhibitors induce dramatic responses in Ewing sarcoma and acute lymphoblastic leukemia when combined with DNA-damaging agents (such as temozolomide). A great need to better understand tumor biology and screening approaches (including PDX models development) still exists, and it can be facilitated through development of immune therapies relevant to the treatment of pediatric sarcoma and through better understanding of the tumor microenvironment. Overcoming the challenges and translating them into opportunities to cure childhood cancer with acceptable quality of life will require a coordinated and global multilevel effort. Many initiatives, such as PPTC, have been committed to these goals for the past 15 years, and a new large-scale international initiative is currently under development to further enhance this effort of developing effective and less toxic treatments for children with cancer.

Dr. Raushan Kurmasheva
Guest Editor

Manuscript Submission Information

Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All submissions that pass pre-check are peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as short communications are invited. For planned papers, a title and short abstract (about 100 words) can be sent to the Editorial Office for announcement on this website.

Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. Journal of Clinical Medicine is an international peer-reviewed open access semimonthly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 2600 CHF (Swiss Francs). Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.

Keywords

  • Childhood cancer
  • Sarcoma
  • Preclinical therapy
  • Chemotherapeutics
  • DNA damage response
  • Synthetic lethality
  • Pediatric preclinical testing program/consortium

Published Papers (6 papers)

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Research

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14 pages, 1497 KiB  
Article
Differentiated Thyroid Cancer in Children in the Last 20 Years: A Regional Study in Romania
by Andreea-Ioana Ștefan, Andra Piciu, Simona Sorana Căinap, Katalin Gabora and Doina Piciu
J. Clin. Med. 2020, 9(11), 3617; https://doi.org/10.3390/jcm9113617 - 10 Nov 2020
Cited by 8 | Viewed by 1707
Abstract
Pediatric thyroid carcinoma is a current area of interest, because there are few studies in this field; the current classification and treatment guidelines are extrapolated from adults, sometimes leading to overly aggressive treatments or incomplete treatment of the disease. The purpose of this [...] Read more.
Pediatric thyroid carcinoma is a current area of interest, because there are few studies in this field; the current classification and treatment guidelines are extrapolated from adults, sometimes leading to overly aggressive treatments or incomplete treatment of the disease. The purpose of this retrospective study is to analyze the presentation, diagnosis, treatment, complications, and outcome of children diagnosed with thyroid cancer in the last two decades (2000–2018) at the Oncological Institute “Prof. Dr. Ion Chiricuță” Cluj-Napoca (IOCN) Romania, a tertiary center in a region with environmental influences from both the nuclear fallout of the Chernobyl accident and from iodine deficiency. The results were compared with those obtained in a previous study carried out in the same institution between 1991–2010, and with those obtained in a similar study carried out in Netherlands between 1970–2013, a cohort of children not exposed to the post-Chernobyl fallout. We statistically analyzed 62 patients. Papillary form was present in the majority of cases, and we highlighted the occurrence of thyroid microcarcinoma in children. A total of 85.4% of patients received radioiodine, the total activity being significantly lower compared with the data from literature. In our study, the prognosis of the disease was excellent, with 100% overall survival. Full article
(This article belongs to the Special Issue The Current Treatment of Childhood Cancer)
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14 pages, 2173 KiB  
Article
Metastatic Burden Defines Clinically and Biologically Distinct Subgroups of Stage 4 High-Risk Neuroblastoma
by Eun Seop Seo, Eun-jin Lee, Boram Lee, Muheon Shin, Young-Seok Cho, Ju Kyung Hyun, Hee Won Cho, Hee Young Ju, Keon Hee Yoo, Hong Hoe Koo, Ji Won Lee and Ki Woong Sung
J. Clin. Med. 2020, 9(9), 2730; https://doi.org/10.3390/jcm9092730 - 24 Aug 2020
Cited by 2 | Viewed by 1998
Abstract
This study aimed to identify the prognostic subgroups of stage 4 high-risk neuroblastoma based on metastatic burden and explore their distinct clinical and genomic features. Patients aged ≥18 months with stage 4 and metaiodobenzylguanidine-avid neuroblastoma were enrolled. One hundred and thirty eligible patients [...] Read more.
This study aimed to identify the prognostic subgroups of stage 4 high-risk neuroblastoma based on metastatic burden and explore their distinct clinical and genomic features. Patients aged ≥18 months with stage 4 and metaiodobenzylguanidine-avid neuroblastoma were enrolled. One hundred and thirty eligible patients were treated under the tandem high-dose chemotherapy scheme. Prognostic significance of metastatic burden measured by the modified Curie score was analyzed using a competing risk approach, and the optimal cut-point was determined. Metastasis-specific subgroups (cut-point: 26) were compared using clinicopathological variables, and differential gene expression analysis and gene set variation analysis (GSVA) were performed using RNA sequencing (RNA-seq). Metastatic burden at diagnosis showed a progressive association with relapse/progression. After applying the cut-point, patients with high metastatic burden showed >3-fold higher risk of relapse/progression than those with low metastatic burden. Moreover, patients with high metastatic burden showed smaller primary tumors and higher biochemical marker levels than those with low metastatic burden. In the genomic analysis, 51 genes were found to be differentially expressed based on the set criteria. GSVA revealed 55 gene sets, which significantly distinguished patients with high metastatic burden from those with low metastatic burden at a false discovery rate <0.25. The results indicated the prognostic significance of metastatic burden in stage 4 high-risk neuroblastoma, and we identified the distinct clinicopathological and genomic features based on metastatic burden. This study may aid in the better understanding and risk-stratification of stage 4 high-risk neuroblastoma patients. Full article
(This article belongs to the Special Issue The Current Treatment of Childhood Cancer)
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Review

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59 pages, 7028 KiB  
Review
Ewing Sarcoma—Diagnosis, Treatment, Clinical Challenges and Future Perspectives
by Stefan K. Zöllner, James F. Amatruda, Sebastian Bauer, Stéphane Collaud, Enrique de Álava, Steven G. DuBois, Jendrik Hardes, Wolfgang Hartmann, Heinrich Kovar, Markus Metzler, David S. Shulman, Arne Streitbürger, Beate Timmermann, Jeffrey A. Toretsky, Yasmin Uhlenbruch, Volker Vieth, Thomas G. P. Grünewald and Uta Dirksen
J. Clin. Med. 2021, 10(8), 1685; https://doi.org/10.3390/jcm10081685 - 14 Apr 2021
Cited by 85 | Viewed by 19506
Abstract
Ewing sarcoma, a highly aggressive bone and soft-tissue cancer, is considered a prime example of the paradigms of a translocation-positive sarcoma: a genetically rather simple disease with a specific and neomorphic-potential therapeutic target, whose oncogenic role was irrefutably defined decades ago. This is [...] Read more.
Ewing sarcoma, a highly aggressive bone and soft-tissue cancer, is considered a prime example of the paradigms of a translocation-positive sarcoma: a genetically rather simple disease with a specific and neomorphic-potential therapeutic target, whose oncogenic role was irrefutably defined decades ago. This is a disease that by definition has micrometastatic disease at diagnosis and a dismal prognosis for patients with macrometastatic or recurrent disease. International collaborations have defined the current standard of care in prospective studies, delivering multiple cycles of systemic therapy combined with local treatment; both are associated with significant morbidity that may result in strong psychological and physical burden for survivors. Nevertheless, the combination of non-directed chemotherapeutics and ever-evolving local modalities nowadays achieve a realistic chance of cure for the majority of patients with Ewing sarcoma. In this review, we focus on the current standard of diagnosis and treatment while attempting to answer some of the most pressing questions in clinical practice. In addition, this review provides scientific answers to clinical phenomena and occasionally defines the resulting translational studies needed to overcome the hurdle of treatment-associated morbidities and, most importantly, non-survival. Full article
(This article belongs to the Special Issue The Current Treatment of Childhood Cancer)
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27 pages, 4039 KiB  
Review
Preclinical In Vivo Modeling of Pediatric Sarcoma—Promises and Limitations
by Roland Imle, Felix K. F. Kommoss and Ana Banito
J. Clin. Med. 2021, 10(8), 1578; https://doi.org/10.3390/jcm10081578 - 08 Apr 2021
Cited by 13 | Viewed by 3512
Abstract
Pediatric sarcomas are an extremely heterogeneous group of genetically distinct diseases. Despite the increasing knowledge on their molecular makeup in recent years, true therapeutic advancements are largely lacking and prognosis often remains dim, particularly for relapsed and metastasized patients. Since this is largely [...] Read more.
Pediatric sarcomas are an extremely heterogeneous group of genetically distinct diseases. Despite the increasing knowledge on their molecular makeup in recent years, true therapeutic advancements are largely lacking and prognosis often remains dim, particularly for relapsed and metastasized patients. Since this is largely due to the lack of suitable model systems as a prerequisite to develop and assess novel therapeutics, we here review the available approaches to model sarcoma in vivo. We focused on genetically engineered and patient-derived mouse models, compared strengths and weaknesses, and finally explored possibilities and limitations to utilize these models to advance both biological understanding as well as clinical diagnosis and therapy. Full article
(This article belongs to the Special Issue The Current Treatment of Childhood Cancer)
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22 pages, 11223 KiB  
Review
Recent Developments in Nanomedicine for Pediatric Cancer
by Shicheng Yang, Mia Wallach, Apurva Krishna, Raushan Kurmasheva and Srinivas Sridhar
J. Clin. Med. 2021, 10(7), 1437; https://doi.org/10.3390/jcm10071437 - 01 Apr 2021
Cited by 18 | Viewed by 4050
Abstract
Cancer is the second biggest cause of death in children in the US. With the development of chemotherapy, there has been a substantial increase in the overall survival rate in the last 30 years. However, the overall mortality rate in children with cancer [...] Read more.
Cancer is the second biggest cause of death in children in the US. With the development of chemotherapy, there has been a substantial increase in the overall survival rate in the last 30 years. However, the overall mortality rate in children with cancer remains 25%, and many survivors experience a decline in overall quality of life and long-term adverse effects caused by treatments. Although cancer cells share common characteristics, pediatric cancers are different from adult cancers in their prevalence, mutation load, and drug response. Therefore, there is an urgent unmet need to develop therapeutic approaches specifically designed for children with cancer. Nanotechnology can potentially overcome the deficiencies of conventional methods of administering chemotherapy and ultimately improve clinical outcomes. The nanoparticle-based drug delivery systems can decrease the toxicity of therapy, provide a sustained or controlled drug release, improve the pharmacokinetic properties of loading contents, and achieve a targeted drug delivery with achievable modifications. Furthermore, therapeutic approaches based on combining nanoformulated drugs with novel immunotherapeutic agents are emerging. In this review, we discussed the recently developed nanotechnology-based strategies for treating blood and solid pediatric cancers. Full article
(This article belongs to the Special Issue The Current Treatment of Childhood Cancer)
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Other

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11 pages, 1247 KiB  
Perspective
Developing New Agents for Treatment of Childhood Cancer: Challenges and Opportunities for Preclinical Testing
by Samson Ghilu, Raushan T. Kurmasheva and Peter J. Houghton
J. Clin. Med. 2021, 10(7), 1504; https://doi.org/10.3390/jcm10071504 - 04 Apr 2021
Cited by 2 | Viewed by 2151
Abstract
Developing new therapeutics for the treatment of childhood cancer has challenges not usually associated with adult malignancies. Firstly, childhood cancer is rare, with approximately 12,500 new diagnoses annually in the U.S. in children 18 years or younger. With current multimodality treatments, the 5-year [...] Read more.
Developing new therapeutics for the treatment of childhood cancer has challenges not usually associated with adult malignancies. Firstly, childhood cancer is rare, with approximately 12,500 new diagnoses annually in the U.S. in children 18 years or younger. With current multimodality treatments, the 5-year event-free survival exceeds 80%, and 70% of patients achieve long-term “cure”, hence the overall number of patients eligible for experimental drugs is small. Childhood cancer comprises many disease entities, the most frequent being acute lymphoblastic leukemias (25% of cancers) and brain tumors (21%), and each of these comprises multiple molecular subtypes. Hence, the numbers of diagnoses even for the more frequently occurring cancers of childhood are small, and undertaking clinical trials remains a significant challenge. Consequently, development of preclinical models that accurately represent each molecular entity can be valuable in identifying those agents or combinations that warrant clinical evaluation. Further, new regulations under the Research to Accelerate Cures and Equity for Children Act (RACE For Children Act) will change the way in which drugs are developed. Here, we will consider some of the limitations of preclinical models and consider approaches that may improve their ability to translate therapy to clinical trial more accurately. Full article
(This article belongs to the Special Issue The Current Treatment of Childhood Cancer)
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