%0 Journal Article %J Am J Hematol %D 2019 %T Idiopathic neutropenia of infancy: Data from the Italian Neutropenia Registry. %A Farruggia, Piero %A Fioredda, Francesca %A Puccio, Giuseppe %A Onofrillo, Daniela %A Russo, Giovanna %A Barone, Angelica %A Bonanomi, Sonia %A Boscarol, Gianluca %A Finocchi, Andrea %A Ghilardi, Roberta %A Giordano, Paola %A Ladogana, Saverio %A Lassandro, Giuseppe %A Luti, Laura %A Lanza, Tiziana %A Mandaglio, Rosalba %A Marra, Nicoletta %A Martire, Baldassare %A Mastrodicasa, Elena %A Motta, Milena %A Notarangelo, Lucia Dora %A Pillon, Marta %A Porretti, Laura %A Serafinelli, Jessica %A Trizzino, Angela %A Tucci, Fabio %A Veltroni, Marinella %A Verzegnassi, Federico %A Ramenghi, Ugo %A Dufour, Carlo %X

Autoimmune neutropenia of infancy (AIN) is characterized by low risk of severe infection, tendency to spontaneously resolve and typically onset at ≤4-5 years of age; it is due to auto-antibodies whose detection is often difficult. In case of negativity of 4 antineutrophils autoantibody tests, after having excluded ethnic, postinfection, drug induced, or congenital neutropenia, according to the Italian guidelines the patients will be defined as affected by "idiopathic neutropenia" (IN). We describe the characteristics of 85 IN patients enrolled in the Italian neutropenia registry: they were compared with 336 children affected by AIN. The 2 groups were clinically very similar and the main differences were detection age (later in IN), length of disease (longer in IN) and, among recovered patients, age of spontaneous recovery: the median age at resolution was 2.13 years in AINs and 3.03 years in INs (P = .00002). At bivariate analysis among AIN patients earlier detection age (P = .00013), male sex (P = .000748), absence of leucopenia (P = .0045), and absence of monocytosis (P = .0419) were significantly associated with earlier recovery; in the IN group only detection age (P = .013) and absence of monocytosis (P = .0333) were significant. At multivariate analysis detection age and absence of monocytosis were independently significant (P = 6.7e-05 and 4.4e-03, respectively) in the AIN group, whereas in the IN group only detection age stayed significant (P = .013).

%B Am J Hematol %V 94 %P 216-222 %8 2019 Feb %G eng %N 2 %1 http://www.ncbi.nlm.nih.gov/pubmed/30456824?dopt=Abstract %R 10.1002/ajh.25353 %0 Journal Article %J Pediatr Blood Cancer %D 2018 %T Multicenter randomized, double-blind controlled trial to evaluate the efficacy of laser therapy for the treatment of severe oral mucositis induced by chemotherapy in children: laMPO RCT. %A Gobbo, Margherita %A Verzegnassi, Federico %A Ronfani, Luca %A Zanon, Davide %A Melchionda, Fraia %A Bagattoni, Simone %A Majorana, Alessandra %A Bardellini, Elena %A Mura, Rosamaria %A Piras, Alessandra %A Petris, Maria Grazia %A Mariuzzi, Maria Livia %A Barone, Angelica %A Merigo, Elisabetta %A Decembrino, Nunzia %A Vitale, Marina Consuelo %A Berger, Massimo %A Defabianis, Patrizia %A Biasotto, Matteo %A Ottaviani, Giulia %A Zanazzo, Giulio Andrea %X

OBJECTIVES: To demonstrate the efficacy of laser photobiomodulation (PBM) compared to that of placebo on severe oral mucositis (OM) in pediatric oncology patients. The primary objective was the reduction of OM grade (World Health Organization [WHO] scale) 7 days after starting PBM. Secondary objectives were reduction of pain, analgesic consumption, and incidence of side effects.

METHODS: One hundred and one children with WHO grade > 2 chemotherapy-induced OM were enrolled in eight Italian hospitals. Patients were randomized to either PBM or sham treatment for four consecutive days (days +1 to +4). On days +4, +7, and +11, OM grade, pain (following a 0-10 numeric pain rating scale, NRS) and need for analgesics were evaluated by an operator blinded to treatment.

RESULTS: Fifty-one patients were allocated to the PBM group, and 50 were allocated to the sham group. In total, 93.7% of PBM patients and 72% of sham patients had OM grade < 3 WHO on day +7 (P = 0.01). A significant reduction of pain was registered on day +7 in the PBM versus sham group (NRS 1 [0-3] vs. 2.5 [1-5], P < 0.006). Reduced use of analgesics was reported in the PBM group, although it was not statistically significant. No significant adverse events attributable to treatment were recorded.

CONCLUSIONS: PBM is a safe, feasible, and effective treatment for children affected by chemotherapy-induced OM, as it accelerates mucosal recovery and reduces pain.

%B Pediatr Blood Cancer %V 65 %P e27098 %8 2018 Aug %G eng %N 8 %1 http://www.ncbi.nlm.nih.gov/pubmed/29727048?dopt=Abstract %R 10.1002/pbc.27098 %0 Journal Article %J Am J Hematol %D 2017 %T Autoimmune neutropenia of childhood secondary to other autoimmune disorders: Data from the Italian neutropenia registry. %A Farruggia, Piero %A Puccio, Giuseppe %A Fioredda, Francesca %A Lanza, Tiziana %A Porretti, Laura %A Ramenghi, Ugo %A Barone, Angelica %A Bonanomi, Sonia %A Finocchi, Andrea %A Ghilardi, Roberta %A Ladogana, Saverio %A Marra, Nicoletta %A Martire, Baldassare %A Notarangelo, Lucia Dora %A Onofrillo, Daniela %A Pillon, Marta %A Russo, Giovanna %A Lo Valvo, Laura %A Serafinelli, Jessica %A Tucci, Fabio %A Zunica, Fiammetta %A Verzegnassi, Federico %A Dufour, Carlo %K Autoimmune Diseases %K Child %K Disease Susceptibility %K Female %K Humans %K Immunoglobulins, Intravenous %K Immunosuppressive Agents %K Infant, Newborn %K Infant, Premature %K Infant, Premature, Diseases %K Italy %K Male %K Neutropenia %K Prevalence %K Registries %B Am J Hematol %V 92 %P E546-E549 %8 2017 Sep %G eng %N 9 %1 http://www.ncbi.nlm.nih.gov/pubmed/28567966?dopt=Abstract %R 10.1002/ajh.24803 %0 Journal Article %J Am J Hematol %D 2016 %T Somatic, hematologic phenotype, long-term outcome, and effect of hematopoietic stem cell transplantation. An analysis of 97 Fanconi anemia patients from the Italian national database on behalf of the Marrow Failure Study Group of the AIEOP (Italian Associ %A Svahn, Johanna %A Bagnasco, Francesca %A Cappelli, Enrico %A Onofrillo, Daniela %A Caruso, Silvia %A Corsolini, Fabio %A De Rocco, Daniela %A Savoia, Anna %A Longoni, Daniela %A Pillon, Marta %A Marra, Nicoletta %A Ramenghi, Ugo %A Farruggia, Piero %A Locasciulli, Anna %A Addari, Carmen %A Cerri, Carla %A Mastrodicasa, Elena %A Casazza, Gabriella %A Verzegnassi, Federico %A Riccardi, Francesca %A Haupt, Riccardo %A Barone, Angelica %A Cesaro, Simone %A Cugno, Chiara %A Dufour, Carlo %X

We analyzed 97 Fanconi anemia patients from a clinic/biological database for genotype, somatic, and hematologic phenotype, adverse hematological events, solid tumors, and treatment. Seventy-two patients belonged to complementation group A. Eighty percent of patients presented with mild/moderate somatic phenotype and most with cytopenia. No correlation was seen between somatic/hematologic phenotype and number of missense mutations of FANCA alleles. Over follow-up, 33% of patients improved or maintained mild/moderate cytopenia or normal blood count, whereas remaining worsened cytopenia. Eleven patients developed a hematological adverse event (MDS, AML, pathological cytogenetics) and three developed solid tumors. 10 years cumulative risk of death of the whole cohort was 25.6% with median follow-up 5.8 years. In patients eligible to hematopoietic stem cell transplantation because of moderate cytopenia, mortality was significantly higher in subjects transplanted from matched unrelated donor over nontransplanted subjects, whereas there was no significant difference between matched sibling donor transplants and nontransplanted patients. In patients eligible to transplant because of severe cytopenia and clonal disease, mortality risk was not significantly different in transplanted from matched unrelated versus matched sibling donor versus nontransplanted subjects. The decision to transplant should rely on various elements including, type of donor, HLA matching, patient comorbidities, impairment, and clonal evolution of hematopoiesis. Am. J. Hematol. 91:666-671, 2016. © 2016 Wiley Periodicals, Inc.

%B Am J Hematol %V 91 %P 666-71 %8 2016 Jul %G eng %N 7 %1 http://www.ncbi.nlm.nih.gov/pubmed/27013026?dopt=Abstract %R 10.1002/ajh.24373 %0 Journal Article %J J Vasc Access %D 2015 %T Central venous access devices in pediatric malignancies: a position paper of Italian Association of Pediatric Hematology and Oncology. %A Crocoli, Alessandro %A Tornesello, Assunta %A Pittiruti, Mauro %A Barone, Angelica %A Muggeo, Paola %A Inserra, Alessandro %A Molinari, Angelo Claudio %A Grillenzoni, Valeria %A Durante, Viviana %A Cicalese, Maria Pia %A Zanazzo, Giulio Andrea %A Cesaro, Simone %X

INTRODUCTION: Treatment of pediatric malignancies is becoming progressively more complex, implying the adoption of multimodal therapies. A reliable, long-lasting venous access represents one of the critical requirements for the success of those treatments. Recent technical innovations-such as minimally invasive procedures for placement, new devices and novel materials-have rapidly spread for clinical use in adult patients, but are still not consistently used in the pediatric population.

METHODS: The Supportive Therapy Working Group of Italian Association of Hematology and Oncology (AIEOP) reviewed medical literature focusing on new aspects of central venous access devices (VADs) in pediatric patients affected by oncohematological diseases.

RESULTS: Appropriate recommendations for clinical use in these patients have been discussed and formulated.

CONCLUSIONS: The importance of the correct choice, management and use of VADs in pediatric oncohematological patients is a necessary prerequisite for an adequate standard of care, also considering the increased chances of cure and the longer life expectancy of those patients with modern therapies.

%B J Vasc Access %V 16 %P 130-6 %8 2015 Mar-Apr %G eng %N 2 %1 http://www.ncbi.nlm.nih.gov/pubmed/25362978?dopt=Abstract %R 10.5301/jva.5000314 %0 Journal Article %J J Pediatr %D 2014 %T Single-day trimethoprim/sulfamethoxazole prophylaxis for Pneumocystis pneumonia in children with cancer. %A Caselli, Désirée %A Petris, Maria Grazia %A Rondelli, Roberto %A Carraro, Francesca %A Colombini, Antonella %A Muggeo, Paola %A Ziino, Ottavio %A Melchionda, Fraia %A Russo, Giovanna %A Pierani, Paolo %A Soncini, Elena %A DeSantis, Raffaella %A Zanazzo, Giulio %A Barone, Angelica %A Cesaro, Simone %A Cellini, Monica %A Mura, Rossella %A Milano, Giuseppe M %A Meazza, Cristina %A Cicalese, Maria P %A Tropia, Serena %A De Masi, Salvatore %A Castagnola, Elio %A Aricò, Maurizio %K Anti-Infective Agents %K Child %K Dose-Response Relationship, Drug %K Drug Administration Schedule %K Follow-Up Studies %K Hematologic Neoplasms %K Humans %K Incidence %K Italy %K Pneumocystis carinii %K Pneumonia, Pneumocystis %K Prospective Studies %K Treatment Outcome %K Trimethoprim, Sulfamethoxazole Drug Combination %X

OBJECTIVE: To determine whether a simplified, 1-day/week regimen of trimethoprim/sulfamethoxazole is sufficient to prevent Pneumocystis (jirovecii [carinii]) pneumonia (PCP). Current recommended regimens for prophylaxis against PCP range from daily administration to 3 consecutive days per week dosing.

STUDY DESIGN: A prospective survey of the regimens adopted for the PCP prophylaxis in all patients treated for childhood cancer at pediatric hematology-oncology centers of the Associazione Italiana Ematologia Oncologia Pediatrica.

RESULTS: The 20 centers participating in the study reported a total of 2466 patients, including 1093 with solid tumor and 1373 with leukemia/lymphoma (or primary immunodeficiency; n = 2). Of these patients, 1371 (55.6%) received the 3-day/week prophylaxis regimen, 406 (16.5%) received the 2-day/week regimen, and 689 (27.9%), including 439 with leukemia/lymphoma, received the 1-day/week regimen. Overall, only 2 cases of PCP (0.08%) were reported, both in the 2-day/week group. By intention to treat, the cumulative incidence of PCP at 3 years was 0.09% overall (95% CI, 0.00-0.40%) and 0.51% for the 2-day/week group (95% CI, 0.10%-2.00%). Remarkably, both patients who failed had withdrawn from prophylaxis.

CONCLUSION: A single-day course of prophylaxis with trimethoprim/sulfamethoxazole may be sufficient to prevent PCP in children with cancer undergoing intensive chemotherapy regimens. This simplified strategy might have implications for the emerging need for PCP prophylaxis in other patients subjected to the increased use of biological and nonbiological agents that induce higher levels of immune suppression, such as those with rheumatic diseases.

%B J Pediatr %V 164 %P 389-92.e1 %8 2014 Feb %G eng %N 2 %1 http://www.ncbi.nlm.nih.gov/pubmed/24252793?dopt=Abstract %R 10.1016/j.jpeds.2013.10.021