Review
Oncology
Muhammad Umair Mushtaq, Moazzam Shahzad, Ezza Tariq, Qamar Iqbal, Sibgha Gull Chaudhary, Muhammad U. Zafar, Iqra Anwar, Nausheen Ahmed, Rajat Bansal, Anurag K. Singh, Sunil H. Abhyankar, Natalie S. Callander, Peiman Hematti, Joseph P. McGuirk
Summary: A systematic review and meta-analysis evaluated the outcomes of mismatched unrelated donor hematopoietic stem cell transplantation (MMUD-HSCT). The results showed that MMUD-HSCT had favorable treatment outcomes and an acceptable toxicity profile in patients lacking HLA-matched or haploidentical donors, thus expanding the accessibility of HSCT to underrepresented populations.
FRONTIERS IN ONCOLOGY
(2022)
Article
Oncology
Bhagirathbhai Dholaria, Myriam Labopin, Jaime Sanz, Annalisa Ruggeri, Jan Cornelissen, Helene Labussiere-Wallet, Didier Blaise, Edouard Forcade, Patrice Chevallier, Anna Grassi, Ludmila Zubarovskaya, Jurgen Kuball, Patrice Ceballos, Fabio Ciceri, Frederic Baron, Bipin N. Savani, Arnon Nagler, Mohamad Mohty
Summary: This study compared the outcomes of CBT and single-allele-mismatched MMUD allo-HCT with PTCy in acute myeloid leukemia. The results showed that CBT was associated with higher incidence of graft failure and non-relapse mortality compared to MMUD, leading to lower leukemia-free survival, overall survival, and GVHD-free, relapse-free survival. In conclusion, MMUD allo-HCT with PTCy may be a preferred choice over CBT for patients without a fully matched donor.
JOURNAL OF HEMATOLOGY & ONCOLOGY
(2021)
Article
Oncology
Gaganvir Parmar, David S. Allan, Gail Morris, Nicholas Dibdin, Kathy Ganz, Karen Mostert, Kristjan Paulson, Tanya Petraszko, Nora Stevens, Matthew D. Seftel
Summary: The COVID-19 pandemic had significant effects on unrelated donor allogeneic peripheral blood stem cell collections, including increased cryopreservation and higher demand for cell doses. Despite these changes, collection targets were still met, but at the expense of increased adverse events in donors and products.
Article
Pediatrics
Chuwen Huang, Yuhua Qu, Sha Liu, Shushan Nie, Hua Jiang
Summary: This study investigated the outcomes of different donor grafts (fully-matched and mismatched) for patients with beta-thalassemia major and concluded that, based on a specific conditioning regimen, mismatched donor graft transplantation could achieve similar results as fully-matched donor graft transplantation.
TRANSLATIONAL PEDIATRICS
(2021)
Article
Oncology
Yue Lu, Min Xiong, Rui-Juan Sun, Jian-Ping Zhang, Yan-Li Zhao, Zhi-Jie Wei, Xing-Yu Cao, Jia-Rui Zhou, De-Yan Liu, Dao-Pei Lu
Summary: In pediatric patients with acquired SAA, UCBD-HSCT showed higher incidence of GF, lower incidence and longer duration of platelet engraftment, and inferior FFS and GRFS compared to HSCT with HID or MUD.
LEUKEMIA & LYMPHOMA
(2022)
Article
Hematology
Allison Barz Leahy, Yimei Li, Julie-An Talano, Caitlin W. Elgarten, Alix E. Seif, Yongping Wang, Bryon Johnson, Dimitri S. Monos, Stephan Kadauke, Timothy S. Olson, Jason Freedman, Lisa Wray, Stephan A. Grupp, Nancy Bunin
Summary: URD TCRαβ/CD19-depleted peripheral HSCT is a safe and effective approach to transplantation for children/YAs with leukemia, reducing the risks of GVHD and relapse.
Review
Medicine, General & Internal
Akshay Sharma, Vanitha A. Jagannath, Latika Puri
Summary: Thalassaemia is an autosomal recessive blood disorder caused by mutations in globin genes, leading to reduced synthesis of certain globin chains. Current treatment involves regular transfusions and iron chelation therapy, with allogeneic hematopoietic stem cell transplantation being the only potentially curative option. Gene therapy for thalassaemia is also emerging as a promising alternative with curative potential.
COCHRANE DATABASE OF SYSTEMATIC REVIEWS
(2021)
Review
Immunology
Scott M. Krummey, Alison J. Gareau
Summary: Advances in hematopoietic stem cell transplant have led to changes in donor selection approach, including the use of haploidentical donors or permissive HLA mismatches. However, this may increase the risk of donor-specific HLA antibodies, which can limit engraftment. Desensitization treatments can effectively reduce HLA antibodies and improve transplant outcomes. The consideration and management of HLA antibodies in donor selection are increasingly important in HSCT.
FRONTIERS IN IMMUNOLOGY
(2022)
Article
Hematology
Curtis Marcoux, David Marin, Jeremy Ramdial, Gheath AlAtrash, Amin M. Alousi, Betul Oran, Partow Kebriaei, Uday R. Popat, Katayoun Rezvani, Richard E. Champlin, Elizabeth J. Shpall, Rohtesh S. Mehta
Summary: Optimal donor selection is crucial in allogeneic hematopoietic cell transplantation, and this study compares the outcomes of patients who received transplants from younger haploidentical donors or older matched unrelated donors. The use of younger haploidentical donors was associated with improved survival and lower rates of GVHD compared to older MUDs. These findings support the use of younger haploidentical donors with post-transplant cyclophosphamide in patients with MDS or AML.
AMERICAN JOURNAL OF HEMATOLOGY
(2023)
Article
Education, Scientific Disciplines
Shukaib Arslan, Monzr M. Al Malki
Summary: This article presents a rational and stepwise approach to the use of MMUD for hematopoietic cell transplant in patients with hematologic malignancies. It also reviews novel approaches to reduce the incidence of graft-versus-host disease and improve transplant outcomes in MMUD recipients.
HEMATOLOGY-AMERICAN SOCIETY OF HEMATOLOGY EDUCATION PROGRAM
(2022)
Article
Oncology
Pietro Crivello, Esteban Arrieta-Bolanos, Meilun He, Tao Wang, Stephanie Fingerson, Shahinaz M. Gadalla, Sophie Paczesny, Steven G. E. Marsh, Stephanie L. J. Lee, Stephen R. Spellman, Yung-Tsi Bolon, Katharina Fleischhauer
Summary: This study found that immunopeptidome divergence is a determinant of T-cell alloreactivity and clinical tolerability in single class I HLA-mismatched unrelated donor hematopoietic cell transplantation. Using hierarchical clustering of peptide-binding motifs, the authors classified 68.1% of the HLA-mismatched transplants as matched or mismatched. Mismatched transplants were associated with lower overall survival.
JOURNAL OF CLINICAL ONCOLOGY
(2023)
Article
Oncology
Neema P. Mayor, Tao Wang, Stephanie J. Lee, Michelle Kuxhausen, Cynthia Vierra-Green, Dominic J. Barker, Jeffrey Auletta, Vijaya R. Bhatt, Shahinaz M. Gadalla, Loren Gragert, Yoshihiro Inamoto, Gerald P. Morris, Sophie Paczesny, Ran Reshef, Olle Ringden, Bronwen E. Shaw, Peter Shaw, Stephen R. Spellman, Steven G. E. Marsh
Summary: This study found that UHR HLA matching may not increase overall survival probability, but it does reduce the risk of aGVHD and in certain subsets, transplant-related mortality. Matching at the UHR level warrants consideration when choosing between multiple 10 out of 10 HLA-matched donors.
JOURNAL OF CLINICAL ONCOLOGY
(2021)
Article
Medicine, General & Internal
Theresa Hahn, Junke Wang, Leah M. Preus, Ezgi Karaesmen, Abbas Rizvi, Alyssa Clay-Gilmour, Qianqian Zhu, Yiwen Wang, Li Yan, Song Liu, Daniel O. Stram, Loreall Pooler, Xin Sheng, Christopher A. Haiman, David Van den Berg, Amy Webb, Guy Brock, Stephen R. Spellman, Kenan Onel, Philip L. McCarthy, Marcelo C. Pasquini, Lara E. Sucheston-Campbell
Summary: Through genome-wide analysis, novel non-HLA genetic loci were found to be associated with 1-year survival after unrelated donor BMT.
Article
Oncology
Yue Lu, Yan-Li Zhao, Min Xiong, Rui-Juan Sun, Xing-Yu Cao, Zhi-Jie Wei, Dao-Pei Lu
Summary: A study on 32 PNH patients undergoing HID or MUD-HSCT showed no significant differences in 3-year OS and FFS between the two groups, but treatment-related mortality had a negative impact on outcomes. HID and MUD-HSCT are feasible and effective options for PNH patients with certain complications.
LEUKEMIA & LYMPHOMA
(2022)
Article
Hematology
Lu Wang, Peiyan Kong, Cheng Zhang, Li Gao, Lidan Zhu, Jia Liu, Shichun Gao, Ting Chen, Huanfeng Liu, Han Yao, Yuqing Liu, Yimei Feng, Lu Zhao, Yuxia Li, Lei Gao, Xi Zhang
Summary: This study compared the outcomes of patients with hematological malignancies who received different preparations of rabbit antithymocyte globulin (ATG) in unrelated donor hematopoietic stem cell transplantation (HSCT). The results showed that the type of ATG preparation had no significant effect on various outcomes, except for a lower risk of extensive chronic graft-versus-host disease (GVHD) and a higher risk of cytomegaloviremia in patients who received ATG-Genzyme (ATG-G). The study suggests that the choice of ATG preparation should be based on the incidence of extensive chronic GVHD, and the posttransplant management strategy should be adjusted accordingly.
ANNALS OF HEMATOLOGY
(2023)
Article
Pediatrics
Tarun Aurora, Audrey Cole, Parul Rai, Paul Lavoie, Carrie Mcivor, Lisa M. Klesges, Guolian Kang, Janaka S. S. Liyanage, Heather M. Brandt, Jane S. Hankins
Summary: This study evaluated the effectiveness of a vaccine strategy bundle in increasing HPV vaccine initiation and completion rates in a specialty clinic setting. By implementing the bundle, which included staff education, provider incentives, offering vaccines in clinics, and verifying vaccine completion, the clinic successfully improved HPV vaccine initiation and completion rates among sickle cell disease patients.
JOURNAL OF PEDIATRICS
(2024)
Article
Pediatrics
Nienke M. Halbmeijer, Wes Onland, Jeroen Dudink, Filip Cools, Anne Debeer, Anton H. van Kaam, Manon J. N. L. Benders, Niek E. van der Aa
Summary: In ventilated infants born preterm, high dose systemic hydrocortisone initiated between 7 and 14 days after birth did not have a significant impact on brain development.
JOURNAL OF PEDIATRICS
(2024)
Article
Pediatrics
Katherine Melton, Jianfang Liu, Hossein Sadeghi, Maureen George, Arlene Smaldone
Summary: This study aims to identify predictors of change in lung function and body weight during health care transition in cystic fibrosis (CF) patients. The study findings highlight the importance of CF RISE program engagement and reducing gaps in care for improving the transition of adolescents and young adults with CF.
JOURNAL OF PEDIATRICS
(2024)
Article
Pediatrics
Laura A. Duckworth, Kimberly A. Sutton, Nurmohammad Shaikh, Jinli Wang, Carla Hall-Moore, Lori R. Holtz, Phillip I. Tarr, Ronald C. Rubenstein
Summary: The study tested the usefulness of various biomarkers as indicators of gut dysfunction in cystic fibrosis (CF) and investigated the repeatability of these measures in individuals over short periods and their correlation with clinical outcomes. The results showed that elevated levels of fLcn2 in individuals with CF may predict worsened pulmonary function.
JOURNAL OF PEDIATRICS
(2024)
Article
Pediatrics
Lindsey Haack, Nikkan Das, Arvind Hoskoppal, Mark Debrunner, Tarek Alsaied, Gaurav Arora
Summary: RAE on ECG has a low positive predictive value for RAE on echocardiogram in previously healthy young patients. The highest yield for RAE on echocardiogram was observed in patients who were <1 year of age, had RAE in the anterior precordial leads, or displayed right ventricular hypertrophy on ECG.
JOURNAL OF PEDIATRICS
(2024)
Article
Pediatrics
Michael A. Padula, Khatija Naing, Tara L. Wenger, Irfan Ahmad, Carl H. Coghill, K. Taylor Wild, S. Alex Rottgers, Cory M. Resnick, Jeffrey Goldstein, Zarmina Ehsan, Donna Watkins, Nicole Deptula, Kuan-Chi Lai, Janet Lioy, Semsa Gogcu, Christopher M. Cielo
Summary: This study describes the spectrum of disease and burden of care in infants with congenital micrognathia. The results show that these infants commonly require surgical intervention and tube feedings, and disparities based on race and among centers were identified.
JOURNAL OF PEDIATRICS
(2024)
Article
Pediatrics
Michael P. Fundora, Manvitha Kalicheti, Guantao Zhao, Kevin O. Maher, Nicoleta Serban
Summary: This study investigated the variation of outpatient opioid prescribing in postoperative pediatric cardiac patients across the US. The results showed that there were significant differences in opioid prescribing by race, ethnicity, sex, and region.
JOURNAL OF PEDIATRICS
(2024)
Article
Pediatrics
Jia Guo, Brooklyn J. Fraser, Leigh Blizzard, Michael D. Schmidt, Terence Dwyer, Alison J. Venn, Costan G. Magnussen
Summary: There is a correlation between childhood and adulthood cardiorespiratory fitness.
JOURNAL OF PEDIATRICS
(2024)
Article
Pediatrics
Nianzhou Xiao, Michelle Starr, Adrienne Stolfi, Gilad Hamdani, Shireen Hashmat, Stefan G. Kiessling, Christina Sethna, Mahmoud Kallash, Robyn Matloff, Robert Woroniecki, Keia Sanderson, Ikuyo Yamaguchi, Stephen D. Cha, Michael G. Semanik, Rahul Chanchlani, Joseph T. Flynn, Mark Mitsnefes
Summary: This multicenter study reports that most infants diagnosed with idiopathic hypertension in the NICU will discontinue antihypertensive treatment within 2 years of discharge. Antenatal steroid treatment is associated with a decreased likelihood of needing antihypertensive therapy for more than 1 year.
JOURNAL OF PEDIATRICS
(2024)
Editorial Material
Pediatrics
Kanwaljit Singh, John Concato, Jonathan M. Davis
JOURNAL OF PEDIATRICS
(2024)
Article
Pediatrics
Yaxing Meng, Harri Niinikoski, Suvi P. Rovio, Brooklyn J. Fraser, Feitong Wu, Antti Jula, Tapani Ronnemaa, Jorma S. A. Viikari, Olli T. Raitakari, Katja Pahkala, Costan G. Magnussen
Summary: This 26-year study shows a correlation between early-life non-HDL-C levels and future levels. Early dietary counseling can reduce the risk of high pediatric non-HDL-C, emphasizing the importance of early interventions in preventing cardiovascular risks.
JOURNAL OF PEDIATRICS
(2024)
Article
Pediatrics
Kelsey A. B. Gastineau, Rebecca Bell, Allison Hanes, Sandra Mckay, Eric Sigel, Filoteia Popescu, Evan C. Sommer, Shari Barkin
Summary: This study aimed to assess the self-reported counseling outcomes for a firearm safe storage counseling training program provided by the American Academy of Pediatrics. The results demonstrated significant improvement in counseling self-efficacy and frequency one month after the training.
JOURNAL OF PEDIATRICS
(2024)
Article
Pediatrics
Kathryn E. K. Berlin, William Scott, Sara Dawson, David Brousseau, Joanne M. Lagatta
Summary: This prospective cohort study aimed to investigate the impact of bronchopulmonary dysplasia (BPD) on the health-related quality of life (HRQL) of infants from NICU hospitalization to one year post-discharge. The study found that lower HRQL during NICU stay was associated with earlier gestational age, postnatal corticosteroid usage, outborn status, and gastrostomy tube placement. Lower HRQL at 3 and 12 months post-discharge was associated with readmissions, home oxygen use, parent-reported difficulty breathing, lower developmental scores, and not playing with other children. Most parents reported similar or improved HRQL after discharge, but parents of infants with respiratory symptoms experienced less improvement. Efforts to improve parent HRQL should focus on respiratory symptoms and social isolation.
JOURNAL OF PEDIATRICS
(2024)
Article
Pediatrics
Garett J. Griffith, Alan P. Wang, Robert I. Liem, Michael R. Carr, Tyler Corson, Kendra Ward
Summary: This study developed reference values for cardiorespiratory fitness in children aged 6-18 years without underlying heart disease, measured by peak oxygen uptake and treadmill time. Fitness levels increased with age in males but not females. Males generally exhibited higher fitness levels compared to females in the same age groups.
JOURNAL OF PEDIATRICS
(2024)
Article
Pediatrics
David S. Liu, Patricia Miller, Anna Rothenberg, Carley Vuillermin, Peter M. Waters, Andrea S. Bauer
Summary: This study aims to determine if children with elbow flexion contracture (EFC) caused by brachial plexus birth injury (BPBI) are more likely to develop shoulder contracture and undergo surgical treatment. A retrospective review was conducted on children under 2 years old with BPBI who presented to a single children's hospital. The results showed that patients with EFC had reduced shoulder range of motion and higher odds of shoulder contracture and surgical treatment. Prompt referral to a BPBI specialty clinic is recommended for evaluation and potential surgery.
JOURNAL OF PEDIATRICS
(2024)
