Publications by authors named "Julia Balaguer"

11 Publications

  • Page 1 of 1

Travel burden and clinical presentation of retinoblastoma: analysis of 1024 patients from 43 African countries and 518 patients from 40 European countries.

Br J Ophthalmol 2020 Sep 15. Epub 2020 Sep 15.

Pediatric Oncology Unit, Hospital Universitario y Politécnico La Fe, Valencia, Spain.

Background: The travel distance from home to a treatment centre, which may impact the stage at diagnosis, has not been investigated for retinoblastoma, the most common childhood eye cancer. We aimed to investigate the travel burden and its impact on clinical presentation in a large sample of patients with retinoblastoma from Africa and Europe.

Methods: A cross-sectional analysis including 518 treatment-naïve patients with retinoblastoma residing in 40 European countries and 1024 treatment-naïve patients with retinoblastoma residing in 43 African countries.

Results: Capture rate was 42.2% of expected patients from Africa and 108.8% from Europe. African patients were older (95% CI -12.4 to -5.4, p<0.001), had fewer cases of familial retinoblastoma (95% CI 2.0 to 5.3, p<0.001) and presented with more advanced disease (95% CI 6.0 to 9.8, p<0.001); 43.4% and 15.4% of Africans had extraocular retinoblastoma and distant metastasis at the time of diagnosis, respectively, compared to 2.9% and 1.0% of the Europeans. To reach a retinoblastoma centre, European patients travelled 421.8 km compared to Africans who travelled 185.7 km (p<0.001). On regression analysis, lower-national income level, African residence and older age (p<0.001), but not travel distance (p=0.19), were risk factors for advanced disease.

Conclusions: Fewer than half the expected number of patients with retinoblastoma presented to African referral centres in 2017, suggesting poor awareness or other barriers to access. Despite the relatively shorter distance travelled by African patients, they presented with later-stage disease. Health education about retinoblastoma is needed for carers and health workers in Africa in order to increase capture rate and promote early referral.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1136/bjophthalmol-2020-316613DOI Listing
September 2020

Global Retinoblastoma Presentation and Analysis by National Income Level.

JAMA Oncol 2020 05;6(5):685-695

Imam Hussein Cancer Center, Karbala, Iraq.

Importance: Early diagnosis of retinoblastoma, the most common intraocular cancer, can save both a child's life and vision. However, anecdotal evidence suggests that many children across the world are diagnosed late. To our knowledge, the clinical presentation of retinoblastoma has never been assessed on a global scale.

Objectives: To report the retinoblastoma stage at diagnosis in patients across the world during a single year, to investigate associations between clinical variables and national income level, and to investigate risk factors for advanced disease at diagnosis.

Design, Setting, And Participants: A total of 278 retinoblastoma treatment centers were recruited from June 2017 through December 2018 to participate in a cross-sectional analysis of treatment-naive patients with retinoblastoma who were diagnosed in 2017.

Main Outcomes And Measures: Age at presentation, proportion of familial history of retinoblastoma, and tumor stage and metastasis.

Results: The cohort included 4351 new patients from 153 countries; the median age at diagnosis was 30.5 (interquartile range, 18.3-45.9) months, and 1976 patients (45.4%) were female. Most patients (n = 3685 [84.7%]) were from low- and middle-income countries (LMICs). Globally, the most common indication for referral was leukocoria (n = 2638 [62.8%]), followed by strabismus (n = 429 [10.2%]) and proptosis (n = 309 [7.4%]). Patients from high-income countries (HICs) were diagnosed at a median age of 14.1 months, with 656 of 666 (98.5%) patients having intraocular retinoblastoma and 2 (0.3%) having metastasis. Patients from low-income countries were diagnosed at a median age of 30.5 months, with 256 of 521 (49.1%) having extraocular retinoblastoma and 94 of 498 (18.9%) having metastasis. Lower national income level was associated with older presentation age, higher proportion of locally advanced disease and distant metastasis, and smaller proportion of familial history of retinoblastoma. Advanced disease at diagnosis was more common in LMICs even after adjusting for age (odds ratio for low-income countries vs upper-middle-income countries and HICs, 17.92 [95% CI, 12.94-24.80], and for lower-middle-income countries vs upper-middle-income countries and HICs, 5.74 [95% CI, 4.30-7.68]).

Conclusions And Relevance: This study is estimated to have included more than half of all new retinoblastoma cases worldwide in 2017. Children from LMICs, where the main global retinoblastoma burden lies, presented at an older age with more advanced disease and demonstrated a smaller proportion of familial history of retinoblastoma, likely because many do not reach a childbearing age. Given that retinoblastoma is curable, these data are concerning and mandate intervention at national and international levels. Further studies are needed to investigate factors, other than age at presentation, that may be associated with advanced disease in LMICs.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1001/jamaoncol.2019.6716DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7047856PMC
May 2020

Intravitreal melphalan therapy for vitreous seeds in retinoblastoma: Implementation and outcomes of a new chemotherapy protocol.

J Oncol Pharm Pract 2020 Dec 17;26(8):1829-1835. Epub 2020 Feb 17.

H.U.P. La Fe, Pharmacy Department, Valencia, Spain.

Retinoblastoma is the most common paediatric ocular tumour, which appears in the retina. Without treatment, retinoblastoma grows and destroys the internal ocular globe architecture, even leading to metastasis. When treated, overall survival is close to 97%, the alkylating drug melphalan being the most extensively used chemotherapeutic agent in localised treatment. The aim of this study is to describe the implementation of a new intravitreal chemotherapy retinoblastoma treatment protocol for children implanting vitreous seeds through intravitreal melphalan injections and to evaluate the patients' health outcomes treated with it. Between December 2014 and July 2018, seven patients were treated with this protocol. They received a mean of 3.3 cycles of intravitreal melphalan with standard doses of 30 mcg per cycle. In the seven eyes treated in our hospital, the response was as expected; three eyes with vitreous seedings (43%) were successfully treated. The main adverse effects presented by all patients were scars at cryogenisation points. In two patients, the appearance of 'salt and pepper' retinopathy was reported. Oncology pharmacists, as part of the treatment team, can provide information about recommended doses, expected adverse effects, stability of preparations, most appropriate method of processing, packaging, and methods of drug administration, to ensure efficacy and especially safety in the administration of these drugs.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1177/1078155220904410DOI Listing
December 2020

Review: Ewing Sarcoma Predisposition.

Pathol Oncol Res 2020 Oct 26;26(4):2057-2066. Epub 2019 Oct 26.

Pediatric Oncology and Hematology Unit, La Fe Hospital, Valencia, Spain.

Ewing sarcoma is a rare tumor developed in bone and soft tissues of children and teenagers. This entity is biologically led by a chromosomal translocation, typically including EWS and FLI1 genes. Little is known about Ewing sarcoma predisposition, although the role of environmental factors, ethnicity and certain polymorphisms on Ewing sarcoma susceptibility has been studied during the last few years. Its prevalence among cancer predisposition syndromes has also been thoroughly examined. This review summarizes the available evidence on predisposing factors involved in Ewing sarcoma susceptibility. On the basis of these data, an integrated approach of the most influential factors on Ewing sarcoma predisposition is proposed.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1007/s12253-019-00765-3DOI Listing
October 2020

Assessment of the dicentric chromosome assay as a biodosimetry tool for more personalized medicine in a case of a high risk neuroblastoma I-mIBG treatment.

Int J Radiat Biol 2019 03 11;95(3):314-320. Epub 2019 Jan 11.

a Radiation Protection Service , Hospital Universitario y Politécnico La Fe , Valencia , Spain.

Purpose: The aims of this study were to estimate the whole - body absorbed - dose with the Dicentric Chromosome Assay (DCA) (biodosimetry) for I - metaiodobenzylguanidine (I - mIBG) therapy for high - risk neuroblastoma, and to obtain an initial correlation with the physical dosimetry calculated as described by the Medical Internal Radiation Dosimetry formalism (MIRD). Together both objectives will aid the optimization of personalized targeted radionuclide therapies.

Material And Methods: A 12 year-old child with relapsed high-risk neuroblastoma was treated with I-mIBG: a first administration with activity <444 MBq/kg was used as a tracer in order to calculate the activity needed in a second administration to achieve a whole body prescribed dose of ∼4 Gy. Blood samples were obtained before and seven days after each administration to analyze the frequency of dicentrics. Moreover, consequent estimations of retained activity were done every few hours from equivalent dose rate measurements at a fixed position, two meters away from the patient, in order to apply the MIRD procedure. Blood samples were also drawn every 2- to -3 days to assess bone marrow toxicity.

Results: For a total activity of 22,867 MBq administered over two phases, both biological and physical dosimetries were performed. The former estimated a whole-body cumulated dose of 3.53 (2.58-4.41) Gy and the latter a total whole-body absorbed dose of 2.32 ± 0.48 Gy. The patient developed thrombocytopenia grade 3 after both infusions and neutropenia grade 3 and grade 4 (based on CTCAE 4.0) during respective phases.

Conclusion: The results indicate a possible correlation between biodosimetry and standard physical dosimetry in I-mIBG treatment for high-risk neuroblastoma. A larger cohort and refinement of the DCA for internal irradiation are needed to define the role of biodosimetry in clinical situations.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1080/09553002.2019.1549755DOI Listing
March 2019

Successful Second-Line Metronomic Temozolomide in Metastatic Paraganglioma: Case Reports and Review of the Literature.

Clin Med Insights Oncol 2018 9;12:1179554918763367. Epub 2018 Apr 9.

Section on Medical Neuroendocrinology, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD, USA.

Metastatic pheochromocytoma and paraganglioma (mPHEO/PGL) are frequently associated with () mutations. Cyclophosphamide-dacarbazine-vincristine (CVD) regimen is recommended as standard chemotherapy for advanced mPHEO/PGL. There is limited evidence to support the role of metronomic schemes (MS) of chemotherapy in mPHEO/PGL treatment. We report 2 patients with -related mPGL who received a regimen consisting of MS temozolomide (TMZ) and high-dose lanreotide after progression on both CVD chemotherapy and high-dose lanreotide. Molecular profiling of the tumor tissue from both patients revealed hypermethylation of the O6-methylguanine-DNA-methyltransferase () promoter. In one patient, progression-free survival was 13 months and the second patient remained under treatment after 27 months of stabilization of metabolic response of his disease. Treatment was well tolerated, and adverse effects were virtually absent. A modification in the scheme of TMZ from standard schemes to MS is safe and feasible and can be considered in patients with progressive mPHEO/PGL refractory to dacarbazine in standard doses.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1177/1179554918763367DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5922490PMC
April 2018

[Li-Fraumeni: Will the detection in families increase the survival of its members?]

An Pediatr (Engl Ed) 2019 Jan 17;90(1):54-55. Epub 2018 Apr 17.

Sección Oncología Infantil, Hospital Universitario y Politécnico la Fe, Valencia, España.

View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1016/j.anpedi.2018.03.009DOI Listing
January 2019

New prognostic markers in neuroblastoma.

Expert Opin Med Diagn 2012 Nov 11;6(6):555-67. Epub 2012 Jul 11.

Department of Pathology, Medical School, University of Valencia , Avda. Blasco Ibañez 15 Valencia 46010 , Spain +34 96 3864146 ; +34 96 3864173 ;

Introduction: The hallmark of neuroblastoma is its clinical and biological heterogeneity, with the likelihood of cure varying widely according to age at diagnosis, extent of disease and tumor biology. We hope this review will be useful for understanding part of the unfamiliar neuroblastoma codex.

Areas Covered: In the first part of this review, the authors summarize the currently used prognostic factors for risk-adapted therapy, with the focus on clinical management of neuroblastoma patients. In the second part, the authors discuss the evolving prognostic factors for future treatment schemes. A search of online medical research databases was undertaken focusing especially on literature published in the last six years.

Expert Opinion: Harnessing the synergy of the various forms of data, including clinical variables and biomarker profiles, would allow mathematical predictive models to be built for the individual patient, which could eventually become molecular targets of specific therapies.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1517/17530059.2012.704018DOI Listing
November 2012

Selective ophthalmic intra-arterial melphalan therapy for advanced retinoblastoma: implementation and outcomes of a new chemotherapy protocol.

J Oncol Pharm Pract 2013 Jun 25;19(2):159-64. Epub 2012 Sep 25.

H. U y P. La Fe, Pharmacy Valencia, Valencia, Spain.

Retinoblastoma is a relatively uncommon childhood tumor. If untreated, RB grows to fill the eye and destroys the ocular globe's internal architecture. Metastatic spread usually begins after the first 6 months, and death occurs within a matter of years. When treated, overall survival rounds 97%, the alkylating drug melphalan being the most extensively used chemotherapeutic agent in localized treatment. In our hospital, pediatric oncologists asked the Pharmacy Department for assessment in order to implement a new chemotherapy protocol for the treatment of advanced intraocular elegible retinoblastoma cases using melphalan administered directly through the ophthalmic artery. In this paper, we describe the protocol implementation carried out by our collaborative interdisciplinary team as well as the clinical outcomes of five cases treated with ophthalmic intra-arterial melphalan therapy. Oncology pharmacists can contribute with their knowledge to the implementation process of new collaborative practice protocols recommending doses, predicting possible adverse effects and assessing about drug stability and elaboration, packaging and administration methods.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1177/1078155212457964DOI Listing
June 2013

Predictive factors of invasion in eyes with retinoblastoma enucleated after eye salvage treatments.

Pediatr Blood Cancer 2009 Mar;52(3):351-6

Department of Oncology, St Jude Children's Research Hospital, Memphis, Tennessee 38105, USA.

Background: The impact of chemotherapy, focal therapies, radiation and co-existing ocular morbidities on histology of eyes with retinoblastoma enucleated following chemoreduction is not well known.

Procedure: Twenty-five eyes (23 patients) with retinoblastoma enucleated after failing eye-salvage therapy were evaluated. Reasons for enucleation (tumor progression, subretinal or vitreous seeds) and co-morbid conditions (neovascular glaucoma, cataract, vitreous hemorrhage and retinal detachment) were documented. All specimens were reviewed for evidence of ciliary body, choroidal, optic nerve, and scleral invasion.

Results: The median age at diagnosis was 14 months (range, 1-37 months). Twenty eyes were classified as Reese-Ellsworth Group IV-V at diagnosis. Twenty-four eyes had recurrent disease at enucleation; one eye was enucleated for neovascular glaucoma and vitreous hemorrhage. Co-existing ocular morbidities at enucleation included vitreous hemorrhage (n = 6), retinal detachment (n = 9), neovascular glaucoma (n = 9) and cataracts (n = 3). Histologic findings included choroidal invasion (n = 7), ciliary body invasion (n = 4), optic nerve invasion (n = 6) and scleral invasion (n = 3). The median time from diagnosis to enucleation was 11 months. Co-existing retinal detachment and vitreous hemorrhage significantly increased the likelihood of optic nerve invasion (P = 0.014 and P = 0.011, respectively). Prolonged time to enucleation was significantly associated with the likelihood of choroidal (P = 0.010) and ciliary body (P = 0.021) invasion as well as invasion of multiple sites.

Conclusion: In eyes with retinoblastoma enucleated after chemoreduction, co-existing ocular morbidities and time to enucleation are predictive of extra-retinal extension.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1002/pbc.21845DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4643656PMC
March 2009

Pediatric renal adenocarcinoma: a review of our series.

Urology 2008 Oct 27;72(4):790-3. Epub 2008 May 27.

Department of Urology, La Fe Children's Hospital, Valencia, Spain.

Objectives: To report our experience with pediatric renal adenocarcinoma.

Methods: We conducted a retrospective review of renal adenocarcinoma cases from 1971 to 2006.

Results: We treated 154 patients with renal tumor, 4 (2.6%) of whom had adenocarcinoma. The mean age of presentation was 8.5 years (range, 2-15 years). Three patients were female and 1 was male. The most common histologic subtype was clear cell carcinoma. Three patients were diagnosed as stage IIIB and one as stage II, according to Robson's classification. All patients were treated with radical nephroureterectomy. One patient died of disease and the other 3 are currently disease-free.

Conclusions: Adenocarcinoma is an uncommon histopathologic type of pediatric renal tumor. In patients with hematuria and abdominal or flank pain, we should consider this tumor as a possible diagnosis.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1016/j.urology.2008.03.030DOI Listing
October 2008
-->