ABSTRACT A 12-year-old male being treated for a high-risk relapsed T-acute lymphoblastic leukemia... more ABSTRACT A 12-year-old male being treated for a high-risk relapsed T-acute lymphoblastic leukemia presented progressive weakness and numbness of both legs after having received a chemotherapy regimen that included bortezomib. Diagnosis of acute Guillain-Barré syndrome-like inflammatory demyelinating polyneuropathy was made following clinical examination, cerebrospinal fluid analysis, electrodiagnostic studies, magnetic resonance imaging, and serum immunoglobulin antibodies to anti-ganglioside. Intravenous immunoglobulin treatment was started, resulting in complete clinical recovery. Although in rare cases, Guillain-Barré syndrome after bortezomib therapy has been reported; this paper suggests that GBS may occur when bortezomib is administered and high‑dose intravenous immunoglobulin lead to a resolution of the symptoms.
ABSTRACT Leukemia is one of the leading journals in hematology and oncology. It is published mont... more ABSTRACT Leukemia is one of the leading journals in hematology and oncology. It is published monthly and covers all aspects of the research and treatment of leukemia and allied diseases. Studies of normal hemopoiesis are covered because of their comparative relevance.
IntroductionIn Europe, despite recent advances in clinical development, most of the drugs current... more IntroductionIn Europe, despite recent advances in clinical development, most of the drugs currently used to treat childhood cancers are adult medicines, prescribed outside of the authorized indication. In this context, a monocentric retrospective cohort analysis was conducted, evaluating pediatric, adolescent, and young adult patients affected by onco‐hematologic disease, treated with targeted therapies used off‐label or as compassionate use.MethodsThe analysis was conducted on 45 patients aged less than or equal to 30 years with cancer, having received at least one targeted therapy prescribed as off‐label or compassionate use at a large Italian pediatric center between January 1, 2016 and June 30, 2021. Data collected included information on the patient and tumor, data on off‐label/compassionate treatment, and data on safety and efficacy.ResultsTotal 25 out of 45 patients treated with off‐label or compassionate targeted therapies were affected by onco‐hematological diseases. Overall, 22 out of the 52 agents (42%) were prescribed in patients with relapsed neoplasm and 39% (20/52) in patients with refractory diseases. Complete response was observed in more than half (27/52) of treatments. At least one adverse reaction occurred in 76% (n = 22) of agents administered to patients with onco‐hematological tumor and in 43% (n = 10) of agents prescribed to patients with solid tumor.ConclusionThis work aims to provide a snapshot of off‐label and compassionate use prescriptions in a large Italian pediatric cancer center. This study confirms that targeted agents for unauthorized indications are often prescribed in pediatric patients with cancer, especially after disease relapse and that these treatments are mostly tolerable and effective.
In the maintenance phase of Associazione Italiana di Ematologia e Oncologia Pediatrica (AIEOP)‐ B... more In the maintenance phase of Associazione Italiana di Ematologia e Oncologia Pediatrica (AIEOP)‐ Berlin‐Frankfurt‐Muenster (BFM) acute lymphoblastic leukemia (ALL) 2009 protocol, mercaptopurine (MP) is given at the planned dose of 50 mg/m2/day; however, dose adjustments are routinely performed to target patients' white blood cells to the optimal range of 2,000–3,000 cells/μL. Pediatric patients with ALL (n = 290, age: median (1st–3rd quartile): 4.8 (3.0–8.1) years; boys: 56.9%) were enrolled mainly in 4 medium‐large Italian pediatric hospitals; 14.1% of patients relapsed after a median (1st–3rd quartile) follow‐up time of 4.43 (3.82–5.46) years from maintenance beginning. MP metabolites (thionucleotide (TGN) and methyl‐derivatives (MMPN)) were measured in the erythrocytes of 387 blood samples of 200 patients by high performance liquid chromatography with ultraviolet detection. Single‐nucleotide polymorphisms (SNPs; (rs1800462, rs1800460, and rs1142345 in TPMT gene, rs116855232 in...
10017 Background: Bosutinib is a tyrosine kinase inhibitor (TKI), approved for adults with Philad... more 10017 Background: Bosutinib is a tyrosine kinase inhibitor (TKI), approved for adults with Philadelphia Chromosome (Ph+) CML; at the standard initial dose of 400 mg/day in ND patients, and 500 mg/day in resistant/intolerant (R/I) patients, administered orally once daily (QD) with food. Compared to the TKIs already approved in pediatrics, bosutinib has a different tolerability profile, and preclinical data suggest that longitudinal growth is potentially less impaired. Study NCT04258943 is an international, open-label, phase I/II trial, sponsored by the Erasmus Medical Center and the Children's Oncology Group, and funded by Pfizer Inc. The phase II arm enrolled ND patients, as well as R/I ones. The latter are not included in this analysis. Methods: ND patients aged 1-18 years with chronic phase CML, without evidence for organ toxicities, were enrolled. Main exclusion criteria included known T315I or V299L BCR-ABL1 mutations, and use of proton pump inhibitors and CYP3A inducers/inh...
Background Infant leukemia is a rare form of acute leukemia diagnosed prior to 1 year of age with... more Background Infant leukemia is a rare form of acute leukemia diagnosed prior to 1 year of age with an extremely poor prognosis, due to its poor response to current therapies. It comprises about 4% of childhood acute lymphoblastic leukemia (ALL). Isolated initial cutaneous involvement in ALL is very uncommon, and even more in infant ALL. Case Presentation Here we present a case of 2-month-old infant, presenting only nodular skin infiltrates on the scalp with the diagnosis of infant acute lymphoblastic leukemia (ALL), characterized by the immunophenotype of the most immature B-cell precursors (pro-B ALL) and chromosomal translocation t (9;11), associated with the rearrangement of KMTLA2 and AF9 genes, that is a negative prognostic factor. She underwent hematopoietic stem cell transplantation (HSCT) and she is still in remission Conclusions This represents a peculiar case because isolated initial cutaneous involvement in ALL is rare. In fact, most reports of ALL leukemia cutis in litera...
IntroductionIn Europe, despite recent advances in clinical development, most of the drugs current... more IntroductionIn Europe, despite recent advances in clinical development, most of the drugs currently used to treat childhood cancers are adult medicines, prescribed outside of the authorized indication. In this context, a monocentric retrospective cohort analysis was conducted, evaluating pediatric, adolescent, and young adult patients affected by onco‐hematologic disease, treated with targeted therapies used off‐label or as compassionate use.MethodsThe analysis was conducted on 45 patients aged less than or equal to 30 years with cancer, having received at least one targeted therapy prescribed as off‐label or compassionate use at a large Italian pediatric center between January 1, 2016 and June 30, 2021. Data collected included information on the patient and tumor, data on off‐label/compassionate treatment, and data on safety and efficacy.ResultsTotal 25 out of 45 patients treated with off‐label or compassionate targeted therapies were affected by onco‐hematological diseases. Overal...
An exercise program (EP) during cancer treatment seems to be a valid strategy against physiologic... more An exercise program (EP) during cancer treatment seems to be a valid strategy against physiological and quality-of-life impairments, but scientific evidence of benefits among pediatric patients is still limited. This review summarizes the literature focused on randomized controlled trials of EP offered to patients during leukemia and lymphoma treatment. Studies published up to June 2017 were selected from multiple databases and assessed by three independent reviewers for methodological validity. The review identified eight studies, but several types of bias have to be avoided to provide evidence-based recommendations accessible to patients, families, and professionals.
ABSTRACT A 12-year-old male being treated for a high-risk relapsed T-acute lymphoblastic leukemia... more ABSTRACT A 12-year-old male being treated for a high-risk relapsed T-acute lymphoblastic leukemia presented progressive weakness and numbness of both legs after having received a chemotherapy regimen that included bortezomib. Diagnosis of acute Guillain-Barré syndrome-like inflammatory demyelinating polyneuropathy was made following clinical examination, cerebrospinal fluid analysis, electrodiagnostic studies, magnetic resonance imaging, and serum immunoglobulin antibodies to anti-ganglioside. Intravenous immunoglobulin treatment was started, resulting in complete clinical recovery. Although in rare cases, Guillain-Barré syndrome after bortezomib therapy has been reported; this paper suggests that GBS may occur when bortezomib is administered and high‑dose intravenous immunoglobulin lead to a resolution of the symptoms.
ABSTRACT Leukemia is one of the leading journals in hematology and oncology. It is published mont... more ABSTRACT Leukemia is one of the leading journals in hematology and oncology. It is published monthly and covers all aspects of the research and treatment of leukemia and allied diseases. Studies of normal hemopoiesis are covered because of their comparative relevance.
IntroductionIn Europe, despite recent advances in clinical development, most of the drugs current... more IntroductionIn Europe, despite recent advances in clinical development, most of the drugs currently used to treat childhood cancers are adult medicines, prescribed outside of the authorized indication. In this context, a monocentric retrospective cohort analysis was conducted, evaluating pediatric, adolescent, and young adult patients affected by onco‐hematologic disease, treated with targeted therapies used off‐label or as compassionate use.MethodsThe analysis was conducted on 45 patients aged less than or equal to 30 years with cancer, having received at least one targeted therapy prescribed as off‐label or compassionate use at a large Italian pediatric center between January 1, 2016 and June 30, 2021. Data collected included information on the patient and tumor, data on off‐label/compassionate treatment, and data on safety and efficacy.ResultsTotal 25 out of 45 patients treated with off‐label or compassionate targeted therapies were affected by onco‐hematological diseases. Overall, 22 out of the 52 agents (42%) were prescribed in patients with relapsed neoplasm and 39% (20/52) in patients with refractory diseases. Complete response was observed in more than half (27/52) of treatments. At least one adverse reaction occurred in 76% (n = 22) of agents administered to patients with onco‐hematological tumor and in 43% (n = 10) of agents prescribed to patients with solid tumor.ConclusionThis work aims to provide a snapshot of off‐label and compassionate use prescriptions in a large Italian pediatric cancer center. This study confirms that targeted agents for unauthorized indications are often prescribed in pediatric patients with cancer, especially after disease relapse and that these treatments are mostly tolerable and effective.
In the maintenance phase of Associazione Italiana di Ematologia e Oncologia Pediatrica (AIEOP)‐ B... more In the maintenance phase of Associazione Italiana di Ematologia e Oncologia Pediatrica (AIEOP)‐ Berlin‐Frankfurt‐Muenster (BFM) acute lymphoblastic leukemia (ALL) 2009 protocol, mercaptopurine (MP) is given at the planned dose of 50 mg/m2/day; however, dose adjustments are routinely performed to target patients' white blood cells to the optimal range of 2,000–3,000 cells/μL. Pediatric patients with ALL (n = 290, age: median (1st–3rd quartile): 4.8 (3.0–8.1) years; boys: 56.9%) were enrolled mainly in 4 medium‐large Italian pediatric hospitals; 14.1% of patients relapsed after a median (1st–3rd quartile) follow‐up time of 4.43 (3.82–5.46) years from maintenance beginning. MP metabolites (thionucleotide (TGN) and methyl‐derivatives (MMPN)) were measured in the erythrocytes of 387 blood samples of 200 patients by high performance liquid chromatography with ultraviolet detection. Single‐nucleotide polymorphisms (SNPs; (rs1800462, rs1800460, and rs1142345 in TPMT gene, rs116855232 in...
10017 Background: Bosutinib is a tyrosine kinase inhibitor (TKI), approved for adults with Philad... more 10017 Background: Bosutinib is a tyrosine kinase inhibitor (TKI), approved for adults with Philadelphia Chromosome (Ph+) CML; at the standard initial dose of 400 mg/day in ND patients, and 500 mg/day in resistant/intolerant (R/I) patients, administered orally once daily (QD) with food. Compared to the TKIs already approved in pediatrics, bosutinib has a different tolerability profile, and preclinical data suggest that longitudinal growth is potentially less impaired. Study NCT04258943 is an international, open-label, phase I/II trial, sponsored by the Erasmus Medical Center and the Children's Oncology Group, and funded by Pfizer Inc. The phase II arm enrolled ND patients, as well as R/I ones. The latter are not included in this analysis. Methods: ND patients aged 1-18 years with chronic phase CML, without evidence for organ toxicities, were enrolled. Main exclusion criteria included known T315I or V299L BCR-ABL1 mutations, and use of proton pump inhibitors and CYP3A inducers/inh...
Background Infant leukemia is a rare form of acute leukemia diagnosed prior to 1 year of age with... more Background Infant leukemia is a rare form of acute leukemia diagnosed prior to 1 year of age with an extremely poor prognosis, due to its poor response to current therapies. It comprises about 4% of childhood acute lymphoblastic leukemia (ALL). Isolated initial cutaneous involvement in ALL is very uncommon, and even more in infant ALL. Case Presentation Here we present a case of 2-month-old infant, presenting only nodular skin infiltrates on the scalp with the diagnosis of infant acute lymphoblastic leukemia (ALL), characterized by the immunophenotype of the most immature B-cell precursors (pro-B ALL) and chromosomal translocation t (9;11), associated with the rearrangement of KMTLA2 and AF9 genes, that is a negative prognostic factor. She underwent hematopoietic stem cell transplantation (HSCT) and she is still in remission Conclusions This represents a peculiar case because isolated initial cutaneous involvement in ALL is rare. In fact, most reports of ALL leukemia cutis in litera...
IntroductionIn Europe, despite recent advances in clinical development, most of the drugs current... more IntroductionIn Europe, despite recent advances in clinical development, most of the drugs currently used to treat childhood cancers are adult medicines, prescribed outside of the authorized indication. In this context, a monocentric retrospective cohort analysis was conducted, evaluating pediatric, adolescent, and young adult patients affected by onco‐hematologic disease, treated with targeted therapies used off‐label or as compassionate use.MethodsThe analysis was conducted on 45 patients aged less than or equal to 30 years with cancer, having received at least one targeted therapy prescribed as off‐label or compassionate use at a large Italian pediatric center between January 1, 2016 and June 30, 2021. Data collected included information on the patient and tumor, data on off‐label/compassionate treatment, and data on safety and efficacy.ResultsTotal 25 out of 45 patients treated with off‐label or compassionate targeted therapies were affected by onco‐hematological diseases. Overal...
An exercise program (EP) during cancer treatment seems to be a valid strategy against physiologic... more An exercise program (EP) during cancer treatment seems to be a valid strategy against physiological and quality-of-life impairments, but scientific evidence of benefits among pediatric patients is still limited. This review summarizes the literature focused on randomized controlled trials of EP offered to patients during leukemia and lymphoma treatment. Studies published up to June 2017 were selected from multiple databases and assessed by three independent reviewers for methodological validity. The review identified eight studies, but several types of bias have to be avoided to provide evidence-based recommendations accessible to patients, families, and professionals.
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Papers by Nicoletta Bertorello