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  1. Article ; Online: Tyrosine Kinase Inhibitors and Beyond for Chronic Myeloid Leukemia in Children.

    Phillips, Lia N / Hijiya, Nobuko

    Paediatric drugs

    2021  Volume 23, Issue 3, Page(s) 241–251

    Abstract: Chronic myeloid leukemia (CML) is rare in children but presents a unique challenge as recent drug innovations have turned CML into a chronic disease with implications for treatment into adulthood. With the approval of newer-generation tyrosine kinase ... ...

    Abstract Chronic myeloid leukemia (CML) is rare in children but presents a unique challenge as recent drug innovations have turned CML into a chronic disease with implications for treatment into adulthood. With the approval of newer-generation tyrosine kinase inhibitors (TKIs) in addition to imatinib, providers have more options for the treatment of chronic-phase CML (CML-CP) in children. The second-generation TKIs approved for use in children, nilotinib and dasatinib, have higher response rates than first-generation imatinib; however, overall survival rates appear to be the same. Even more options may soon become available with ongoing investigations into the use of bosutinib and ponatinib and other new agents in children. Possible long-term side effects of TKIs, including growth failure, should be carefully acknowledged by the treating provider. Although these known associations may not preclude treatment, providers should be aware of them to guide their management of pediatric patients with CML being treated long term with TKI therapy. Treatment-free remission is a desired goal for pediatric patients and providers alike, but current recommendations are for attempts at achieving this to be restricted to clinical study settings.
    MeSH term(s) Adolescent ; Child ; Child, Preschool ; Humans ; Leukemia, Myelogenous, Chronic, BCR-ABL Positive/drug therapy ; Leukemia, Myelogenous, Chronic, BCR-ABL Positive/pathology ; Protein Kinase Inhibitors/pharmacology ; Protein Kinase Inhibitors/therapeutic use
    Chemical Substances Protein Kinase Inhibitors
    Language English
    Publishing date 2021-04-26
    Publishing country Switzerland
    Document type Journal Article ; Review
    ZDB-ID 1492748-2
    ISSN 1179-2019 ; 1174-5878
    ISSN (online) 1179-2019
    ISSN 1174-5878
    DOI 10.1007/s40272-021-00446-2
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  2. Article ; Online: Iron Deficiency in Chronic Pediatric Heart Failure: Overall Assessment and Outcomes in Dilated Cardiomyopathy.

    Phillips, Lia / Richmond, Marc / Neunert, Cindy / Jin, Zhezhen / Brittenham, Gary M

    The Journal of pediatrics

    2023  Volume 263, Page(s) 113721

    Abstract: Objective: To evaluate the frequency of iron status assessment in pediatric heart failure and the prevalence and adverse effects of absolute iron deficiency in dilated cardiomyopathy-induced heart failure.: Study design: We retrospectively reviewed ... ...

    Abstract Objective: To evaluate the frequency of iron status assessment in pediatric heart failure and the prevalence and adverse effects of absolute iron deficiency in dilated cardiomyopathy-induced heart failure.
    Study design: We retrospectively reviewed records of children with chronic heart failure at our center between 2010 and 2020. In children with dilated cardiomyopathy, we analyzed baseline cardiac function, hemoglobin level, and subsequent risk of composite adverse events (CAE), including death, heart transplant, ventricular assist device (VAD) placement, and transplant registry listing. Absolute iron deficiency and iron sufficiency were defined as transferrin saturations <20% and ≥30%, respectively; and indeterminant iron status as 20%-29%.
    Results: Of 799 patients with chronic heart failure, 471 (59%) had no iron-related laboratory measurements. Of 68 children with dilated cardiomyopathy, baseline transferrin saturation, and quantitative left ventricular ejection fraction (LVEF), 33 (49%) and 14 (21%) were iron deficient and sufficient, respectively, and 21 (31%) indeterminant. LVEF was reduced to 23.6 ± 12.1% from 32.9 ± 16.8% in iron deficiency and sufficiency, respectively (P = .04), without a significant difference in hemoglobin. After stratification by New York Heart Association classification, in advanced class IV, hemoglobin was reduced to 10.9 ± 1.3 g/dL vs 12.7 ± 2.0 g/dL in iron deficiency and sufficiency, respectively (P = .01), without a significant difference in LVEF.
    Conclusions: In this single-center study, iron deficiency was not monitored in most children with chronic heart failure. In pediatric dilated cardiomyopathy-induced heart failure, absolute iron deficiency was prevalent and associated with clinically consequential and possibly correctable decreases in cardiac function and hemoglobin concentration.
    MeSH term(s) Humans ; Child ; Cardiomyopathy, Dilated/complications ; Cardiomyopathy, Dilated/therapy ; Stroke Volume ; Retrospective Studies ; Ventricular Function, Left ; Heart Failure/complications ; Heart Failure/therapy ; Iron Deficiencies ; Iron/pharmacology ; Chronic Disease ; Hemoglobins ; Transferrins/pharmacology
    Chemical Substances Iron (E1UOL152H7) ; Hemoglobins ; Transferrins
    Language English
    Publishing date 2023-09-09
    Publishing country United States
    Document type Journal Article
    ZDB-ID 3102-1
    ISSN 1097-6833 ; 0022-3476
    ISSN (online) 1097-6833
    ISSN 0022-3476
    DOI 10.1016/j.jpeds.2023.113721
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  3. Article ; Online: Successful management of SARS-CoV-2 acute respiratory distress syndrome and newly diagnosed acute lymphoblastic leukemia.

    Phillips, Lia / Pavisic, Jovana / Kaur, Dominder / Dorrello, N Valerio / Broglie, Larisa / Hijiya, Nobuko

    Blood advances

    2020  Volume 4, Issue 18, Page(s) 4358–4361

    MeSH term(s) Adolescent ; Allopurinol/therapeutic use ; Anemia, Hemolytic, Autoimmune/etiology ; Anti-Bacterial Agents/therapeutic use ; Antineoplastic Combined Chemotherapy Protocols/therapeutic use ; Betacoronavirus ; COVID-19 ; Combined Modality Therapy ; Coronavirus Infections/complications ; Coronavirus Infections/diagnostic imaging ; Cytokine Release Syndrome/etiology ; Daunorubicin/administration & dosage ; Fluid Therapy ; Humans ; Male ; Methylprednisolone/therapeutic use ; Pandemics ; Pneumonia, Viral/complications ; Pneumonia, Viral/diagnostic imaging ; Precursor B-Cell Lymphoblastic Leukemia-Lymphoma/complications ; Precursor B-Cell Lymphoblastic Leukemia-Lymphoma/diagnosis ; Precursor B-Cell Lymphoblastic Leukemia-Lymphoma/drug therapy ; Pyrazoles/therapeutic use ; Respiration, Artificial ; Respiratory Distress Syndrome/diagnostic imaging ; Respiratory Distress Syndrome/drug therapy ; Respiratory Distress Syndrome/etiology ; Respiratory Distress Syndrome/therapy ; SARS-CoV-2 ; Vincristine/administration & dosage
    Chemical Substances Anti-Bacterial Agents ; Pyrazoles ; Vincristine (5J49Q6B70F) ; Allopurinol (63CZ7GJN5I) ; ruxolitinib (82S8X8XX8H) ; Methylprednisolone (X4W7ZR7023) ; Daunorubicin (ZS7284E0ZP)
    Keywords covid19
    Language English
    Publishing date 2020-09-11
    Publishing country United States
    Document type Case Reports ; Journal Article
    ZDB-ID 2915908-8
    ISSN 2473-9537 ; 2473-9529
    ISSN (online) 2473-9537
    ISSN 2473-9529
    DOI 10.1182/bloodadvances.2020002745
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  4. Article ; Online: Ovarian Sertoli-Leydig tumor after bone marrow transplant for sickle cell disease.

    Phillips, Lia Neu / Krishnamurti, Lakshmanan / Rytting, Heather / Olson, Thomas A

    Pediatric blood & cancer

    2018  Volume 65, Issue 11, Page(s) e27367

    Abstract: As bone marrow transplant for sickle cell disease becomes increasingly common, long-term outcomes including secondary malignancies are beginning to be described. Here, we report a case of ovarian Sertoli-Leydig tumor that occurred after allogeneic bone ... ...

    Abstract As bone marrow transplant for sickle cell disease becomes increasingly common, long-term outcomes including secondary malignancies are beginning to be described. Here, we report a case of ovarian Sertoli-Leydig tumor that occurred after allogeneic bone marrow transplant for sickle cell disease.
    MeSH term(s) Anemia, Sickle Cell/surgery ; Bone Marrow Transplantation ; Child ; Female ; Humans ; Ovarian Neoplasms/pathology ; Sertoli-Leydig Cell Tumor/pathology ; Young Adult
    Language English
    Publishing date 2018-07-24
    Publishing country United States
    Document type Case Reports ; Journal Article
    ZDB-ID 2131448-2
    ISSN 1545-5017 ; 1545-5009
    ISSN (online) 1545-5017
    ISSN 1545-5009
    DOI 10.1002/pbc.27367
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  5. Article: Successful management of SARS-CoV-2 acute respiratory distress syndrome and newly diagnosed acute lymphoblastic leukemia

    Phillips, Lia / Pavisic, Jovana / Kaur, Dominder / Dorrello, N Valerio / Broglie, Larisa / Hijiya, Nobuko

    Blood Adv

    Keywords covid19
    Publisher WHO
    Document type Article
    Note WHO #Covidence: #760543
    Database COVID19

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  6. Article ; Online: Global, regional, and national disease burden estimates of acute lower respiratory infections due to respiratory syncytial virus in young children in 2015: a systematic review and modelling study.

    Shi, Ting / McAllister, David A / O'Brien, Katherine L / Simoes, Eric A F / Madhi, Shabir A / Gessner, Bradford D / Polack, Fernando P / Balsells, Evelyn / Acacio, Sozinho / Aguayo, Claudia / Alassani, Issifou / Ali, Asad / Antonio, Martin / Awasthi, Shally / Awori, Juliet O / Azziz-Baumgartner, Eduardo / Baggett, Henry C / Baillie, Vicky L / Balmaseda, Angel /
    Barahona, Alfredo / Basnet, Sudha / Bassat, Quique / Basualdo, Wilma / Bigogo, Godfrey / Bont, Louis / Breiman, Robert F / Brooks, W Abdullah / Broor, Shobha / Bruce, Nigel / Bruden, Dana / Buchy, Philippe / Campbell, Stuart / Carosone-Link, Phyllis / Chadha, Mandeep / Chipeta, James / Chou, Monidarin / Clara, Wilfrido / Cohen, Cheryl / de Cuellar, Elizabeth / Dang, Duc-Anh / Dash-Yandag, Budragchaagiin / Deloria-Knoll, Maria / Dherani, Mukesh / Eap, Tekchheng / Ebruke, Bernard E / Echavarria, Marcela / de Freitas Lázaro Emediato, Carla Cecília / Fasce, Rodrigo A / Feikin, Daniel R / Feng, Luzhao / Gentile, Angela / Gordon, Aubree / Goswami, Doli / Goyet, Sophie / Groome, Michelle / Halasa, Natasha / Hirve, Siddhivinayak / Homaira, Nusrat / Howie, Stephen R C / Jara, Jorge / Jroundi, Imane / Kartasasmita, Cissy B / Khuri-Bulos, Najwa / Kotloff, Karen L / Krishnan, Anand / Libster, Romina / Lopez, Olga / Lucero, Marilla G / Lucion, Florencia / Lupisan, Socorro P / Marcone, Debora N / McCracken, John P / Mejia, Mario / Moisi, Jennifer C / Montgomery, Joel M / Moore, David P / Moraleda, Cinta / Moyes, Jocelyn / Munywoki, Patrick / Mutyara, Kuswandewi / Nicol, Mark P / Nokes, D James / Nymadawa, Pagbajabyn / da Costa Oliveira, Maria Tereza / Oshitani, Histoshi / Pandey, Nitin / Paranhos-Baccalà, Gláucia / Phillips, Lia N / Picot, Valentina Sanchez / Rahman, Mustafizur / Rakoto-Andrianarivelo, Mala / Rasmussen, Zeba A / Rath, Barbara A / Robinson, Annick / Romero, Candice / Russomando, Graciela / Salimi, Vahid / Sawatwong, Pongpun / Scheltema, Nienke / Schweiger, Brunhilde / Scott, J Anthony G / Seidenberg, Phil / Shen, Kunling / Singleton, Rosalyn / Sotomayor, Viviana / Strand, Tor A / Sutanto, Agustinus / Sylla, Mariam / Tapia, Milagritos D / Thamthitiwat, Somsak / Thomas, Elizabeth D / Tokarz, Rafal / Turner, Claudia / Venter, Marietjie / Waicharoen, Sunthareeya / Wang, Jianwei / Watthanaworawit, Wanitda / Yoshida, Lay-Myint / Yu, Hongjie / Zar, Heather J / Campbell, Harry / Nair, Harish

    Lancet (London, England)

    2017  Volume 390, Issue 10098, Page(s) 946–958

    Abstract: Background: We have previously estimated that respiratory syncytial virus (RSV) was associated with 22% of all episodes of (severe) acute lower respiratory infection (ALRI) resulting in 55 000 to 199 000 deaths in children younger than 5 years in 2005. ... ...

    Abstract Background: We have previously estimated that respiratory syncytial virus (RSV) was associated with 22% of all episodes of (severe) acute lower respiratory infection (ALRI) resulting in 55 000 to 199 000 deaths in children younger than 5 years in 2005. In the past 5 years, major research activity on RSV has yielded substantial new data from developing countries. With a considerably expanded dataset from a large international collaboration, we aimed to estimate the global incidence, hospital admission rate, and mortality from RSV-ALRI episodes in young children in 2015.
    Methods: We estimated the incidence and hospital admission rate of RSV-associated ALRI (RSV-ALRI) in children younger than 5 years stratified by age and World Bank income regions from a systematic review of studies published between Jan 1, 1995, and Dec 31, 2016, and unpublished data from 76 high quality population-based studies. We estimated the RSV-ALRI incidence for 132 developing countries using a risk factor-based model and 2015 population estimates. We estimated the in-hospital RSV-ALRI mortality by combining in-hospital case fatality ratios with hospital admission estimates from hospital-based (published and unpublished) studies. We also estimated overall RSV-ALRI mortality by identifying studies reporting monthly data for ALRI mortality in the community and RSV activity.
    Findings: We estimated that globally in 2015, 33·1 million (uncertainty range [UR] 21·6-50·3) episodes of RSV-ALRI, resulted in about 3·2 million (2·7-3·8) hospital admissions, and 59 600 (48 000-74 500) in-hospital deaths in children younger than 5 years. In children younger than 6 months, 1·4 million (UR 1·2-1·7) hospital admissions, and 27 300 (UR 20 700-36 200) in-hospital deaths were due to RSV-ALRI. We also estimated that the overall RSV-ALRI mortality could be as high as 118 200 (UR 94 600-149 400). Incidence and mortality varied substantially from year to year in any given population.
    Interpretation: Globally, RSV is a common cause of childhood ALRI and a major cause of hospital admissions in young children, resulting in a substantial burden on health-care services. About 45% of hospital admissions and in-hospital deaths due to RSV-ALRI occur in children younger than 6 months. An effective maternal RSV vaccine or monoclonal antibody could have a substantial effect on disease burden in this age group.
    Funding: The Bill & Melinda Gates Foundation.
    MeSH term(s) Child, Preschool ; Developing Countries ; Global Health ; Hospital Mortality ; Hospitalization/statistics & numerical data ; Humans ; Incidence ; Infant ; Infant, Newborn ; Models, Statistical ; Respiratory Syncytial Viruses/isolation & purification ; Respiratory Tract Infections/epidemiology ; Risk Factors
    Language English
    Publishing date 2017-07-07
    Publishing country England
    Document type Journal Article ; Review ; Systematic Review
    ZDB-ID 3306-6
    ISSN 1474-547X ; 0023-7507 ; 0140-6736
    ISSN (online) 1474-547X
    ISSN 0023-7507 ; 0140-6736
    DOI 10.1016/S0140-6736(17)30938-8
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