Literature DB >> 28479164

Recommendations for Screening and Management of Late Effects in Patients with Severe Combined Immunodeficiency after Allogenic Hematopoietic Cell Transplantation: A Consensus Statement from the Second Pediatric Blood and Marrow Transplant Consortium International Conference on Late Effects after Pediatric HCT.

Jennifer Heimall1, Rebecca H Buckley2, Jennifer Puck3, Thomas A Fleisher4, Andrew R Gennery5, Elie Haddad6, Benedicte Neven7, Mary Slatter5, Skinner Roderick8, K Scott Baker9, Andrew C Dietz10, Christine Duncan11, Linda M Griffith12, Luigi Notarangelo13, Michael A Pulsipher11, Morton J Cowan3.   

Abstract

Severe combined immunodeficiency (SCID) is effectively treated with hematopoietic cell transplantation (HCT), with overall survival approaching 90% in contemporary reports. However, survivors are at risk for developing late complications because of the variable durability of high-quality immune function, underlying genotype of SCID, comorbidities due to infections in the pretransplantation and post-transplantation periods, and use of conditioning before transplantation. An international group of transplantation experts was convened in 2016 to review the current knowledge of late effects seen in SCID patients after HCT and to develop recommendations for screening and monitoring for late effects. This report provides recommendations for screening and management of pediatric and adult SCID patients treated with HCT.
Copyright © 2017 The American Society for Blood and Marrow Transplantation. All rights reserved.

Entities:  

Keywords:  Bone marrow transplantation; Long-term follow-up guidelines; Severe combined immune deficiency

Mesh：

Year:  2017        PMID： 28479164      PMCID： PMC6015789          DOI： 10.1016/j.bbmt.2017.04.026

Source DB:  PubMed          Journal:  Biol Blood Marrow Transplant        ISSN： 1083-8791            Impact factor:   5.742


  42 in total

1.  Intrinsic defects of B cell function in X-linked severe combined immunodeficiency.

Authors:  H White; A Thrasher; P Veys; C Kinnon; H B Gaspar
Journal:  Eur J Immunol       Date:  2000-03       Impact factor: 5.532

2.  Hematopoietic stem cell transplantation for severe combined immunodeficiency in the neonatal period leads to superior thymic output and improved survival.

Authors:  Laurie A Myers; Dhavalkumar D Patel; Jennifer M Puck; Rebecca H Buckley
Journal:  Blood       Date:  2002-02-01       Impact factor: 22.113

3.  National Cancer Institute, National Heart, Lung and Blood Institute/Pediatric Blood and Marrow Transplantation Consortium First International Consensus Conference on late effects after pediatric hematopoietic cell transplantation: the need for pediatric-specific long-term follow-up guidelines.

Authors:  Michael A Pulsipher; Roderick Skinner; George B McDonald; Sangeeta Hingorani; Saro H Armenian; Kenneth R Cooke; Clarisa Gracia; Anna Petryk; Smita Bhatia; Nancy Bunin; Michael L Nieder; Christopher C Dvorak; Lillian Sung; Jean E Sanders; Joanne Kurtzberg; K Scott Baker
Journal:  Biol Blood Marrow Transplant       Date:  2012-01-14       Impact factor: 5.742

4.  NCI, NHLBI/PBMTC First International Conference on Late Effects after Pediatric Hematopoietic Cell Transplantation: health-related quality of life, functional, and neurocognitive outcomes.

Authors:  Susan K Parsons; Sean Phipps; Lillian Sung; K Scott Baker; Michael A Pulsipher; Kirsten K Ness
Journal:  Biol Blood Marrow Transplant       Date:  2011-12-10       Impact factor: 5.742

Review 5.  Recommendations for live viral and bacterial vaccines in immunodeficient patients and their close contacts.

Authors:  William T Shearer; Thomas A Fleisher; Rebecca H Buckley; Zuhair Ballas; Mark Ballow; R Michael Blaese; Francisco A Bonilla; Mary Ellen Conley; Charlotte Cunningham-Rundles; Alexandra H Filipovich; Ramsay Fuleihan; Erwin W Gelfand; Vivian Hernandez-Trujillo; Steven M Holland; Richard Hong; Howard M Lederman; Harry L Malech; Stephen Miles; Luigi D Notarangelo; Hans D Ochs; Jordan S Orange; Jennifer M Puck; John M Routes; E Richard Stiehm; Kathleen Sullivan; Troy Torgerson; Jerry Winkelstein
Journal:  J Allergy Clin Immunol       Date:  2014-02-28       Impact factor: 10.793

6.  Human adenylate kinase 2 deficiency causes a profound hematopoietic defect associated with sensorineural deafness.

Authors:  Chantal Lagresle-Peyrou; Emmanuelle M Six; Capucine Picard; Frédéric Rieux-Laucat; Vincent Michel; Andrea Ditadi; Corinne Demerens-de Chappedelaine; Estelle Morillon; Françoise Valensi; Karen L Simon-Stoos; James C Mullikin; Lenora M Noroski; Céline Besse; Nicolas M Wulffraat; Alina Ferster; Manuel M Abecasis; Fabien Calvo; Christine Petit; Fabio Candotti; Laurent Abel; Alain Fischer; Marina Cavazzana-Calvo
Journal:  Nat Genet       Date:  2008-11-30       Impact factor: 38.330

7.  Long-term clinical outcome of patients with severe combined immunodeficiency who received related donor bone marrow transplants without pretransplant chemotherapy or post-transplant GVHD prophylaxis.

Authors:  Mary Dell Railey; Yuliya Lokhnygina; Rebecca H Buckley
Journal:  J Pediatr       Date:  2009-10-09       Impact factor: 4.406

8.  Newborn screening for severe combined immunodeficiency and T-cell lymphopenia in California: results of the first 2 years.

Authors:  Antonia Kwan; Joseph A Church; Morton J Cowan; Rajni Agarwal; Neena Kapoor; Donald B Kohn; David B Lewis; Sean A McGhee; Theodore B Moore; E Richard Stiehm; Matthew Porteus; Constantino P Aznar; Robert Currier; Fred Lorey; Jennifer M Puck
Journal:  J Allergy Clin Immunol       Date:  2013-07       Impact factor: 10.793

9.  Cognitive and behavioral abnormalities in children after hematopoietic stem cell transplantation for severe congenital immunodeficiencies.

Authors:  Penny Titman; Elizabeth Pink; Emily Skucek; Katherine O'Hanlon; Tim J Cole; Jane Gaspar; Jinhua Xu-Bayford; Alison Jones; Adrian J Thrasher; E Graham Davies; Paul A Veys; H Bobby Gaspar
Journal:  Blood       Date:  2008-07-21       Impact factor: 22.113

10.  Evidence of innate lymphoid cell redundancy in humans.

Authors:  Frédéric Vély; Vincent Barlogis; Blandine Vallentin; Bénédicte Neven; Christelle Piperoglou; Mikael Ebbo; Thibaut Perchet; Maxime Petit; Nadia Yessaad; Fabien Touzot; Julie Bruneau; Nizar Mahlaoui; Nicolas Zucchini; Catherine Farnarier; Gérard Michel; Despina Moshous; Stéphane Blanche; Arnaud Dujardin; Hergen Spits; Jörg H W Distler; Andreas Ramming; Capucine Picard; Rachel Golub; Alain Fischer; Eric Vivier
Journal:  Nat Immunol       Date:  2016-09-12       Impact factor: 25.606
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  12 in total

Review 1.  Long term outcomes of severe combined immunodeficiency: therapy implications.

Authors:  Jennifer Heimall; Morton J Cowan
Journal:  Expert Rev Clin Immunol       Date:  2017-09-23       Impact factor: 4.473

2.  Pediatric Hematopoietic Cell Transplant Patients Who Survive Critical Illness Frequently Have Significant but Recoverable Decline in Functional Status.

Authors:  Matt S Zinter; Richard Holubkov; Martina A Steurer; Christopher C Dvorak; Christine N Duncan; Anil Sapru; Robert F Tamburro; Patrick S McQuillen; Murray M Pollack
Journal:  Biol Blood Marrow Transplant       Date:  2017-11-08       Impact factor: 5.742

3.  SCID genotype and 6-month posttransplant CD4 count predict survival and immune recovery.

Authors:  Elie Haddad; Brent R Logan; Linda M Griffith; Rebecca H Buckley; Roberta E Parrott; Susan E Prockop; Trudy N Small; Jessica Chaisson; Christopher C Dvorak; Megan Murnane; Neena Kapoor; Hisham Abdel-Azim; Imelda C Hanson; Caridad Martinez; Jack J H Bleesing; Sharat Chandra; Angela R Smith; Matthew E Cavanaugh; Soma Jyonouchi; Kathleen E Sullivan; Lauri Burroughs; Suzanne Skoda-Smith; Ann E Haight; Audrey G Tumlin; Troy C Quigg; Candace Taylor; Blachy J Dávila Saldaña; Michael D Keller; Christine M Seroogy; Kenneth B Desantes; Aleksandra Petrovic; Jennifer W Leiding; David C Shyr; Hélène Decaluwe; Pierre Teira; Alfred P Gillio; Alan P Knutsen; Theodore B Moore; Morris Kletzel; John A Craddock; Victor Aquino; Jeffrey H Davis; Lolie C Yu; Geoffrey D E Cuvelier; Jeffrey J Bednarski; Frederick D Goldman; Elizabeth M Kang; Evan Shereck; Matthew H Porteus; James A Connelly; Thomas A Fleisher; Harry L Malech; William T Shearer; Paul Szabolcs; Monica S Thakar; Mark T Vander Lugt; Jennifer Heimall; Ziyan Yin; Michael A Pulsipher; Sung-Yun Pai; Donald B Kohn; Jennifer M Puck; Morton J Cowan; Richard J O'Reilly; Luigi D Notarangelo
Journal:  Blood       Date:  2018-08-28       Impact factor: 22.113

Review 4.  Consensus approach for the management of severe combined immune deficiency caused by adenosine deaminase deficiency.

Authors:  Donald B Kohn; Michael S Hershfield; Jennifer M Puck; Alessandro Aiuti; Annaliesse Blincoe; H Bobby Gaspar; Luigi D Notarangelo; Eyal Grunebaum
Journal:  J Allergy Clin Immunol       Date:  2018-09-05       Impact factor: 10.793

Review 5.  Immune function in childhood cancer survivors: a Children's Oncology Group review.

Authors:  Gregory M T Guilcher; Linda Rivard; Jennifer T Huang; Nicola A M Wright; Lynette Anderson; Hesham Eissa; Wendy Pelletier; Shanti Ramachandran; Tal Schechter; Ami J Shah; Ken Wong; Eric J Chow
Journal:  Lancet Child Adolesc Health       Date:  2021-02-16

Review 6.  Hematopoietic Stem Cell Transplantation for Primary Immunodeficiencies.

Authors:  Andrew R Gennery; Michael H Albert; Mary A Slatter; Arjan Lankester
Journal:  Front Pediatr       Date:  2019-10-30       Impact factor: 3.418

7.  Subsequent neoplasms and late mortality in children undergoing allogeneic transplantation for nonmalignant diseases.

Authors:  Justine M Kahn; Ruta Brazauskas; Heather R Tecca; Stephanie Bo-Subait; David Buchbinder; Minoo Battiwala; Mary E D Flowers; Bipin N Savani; Rachel Phelan; Larisa Broglie; Allistair A Abraham; Amy K Keating; Andrew Daly; Baldeep Wirk; Biju George; Blanche P Alter; Celalettin Ustun; Cesar O Freytes; Amer M Beitinjaneh; Christine Duncan; Edward Copelan; Gerhard C Hildebrandt; Hemant S Murthy; Hillard M Lazarus; Jeffery J Auletta; Kasiani C Myers; Kirsten M Williams; Kristin M Page; Lynda M Vrooman; Maxim Norkin; Michael Byrne; Miguel Angel Diaz; Naynesh Kamani; Neel S Bhatt; Andrew Rezvani; Nosha Farhadfar; Parinda A Mehta; Peiman Hematti; Peter J Shaw; Rammurti T Kamble; Raquel Schears; Richard F Olsson; Robert J Hayashi; Robert Peter Gale; Samantha J Mayo; Saurabh Chhabra; Seth J Rotz; Sherif M Badawy; Siddhartha Ganguly; Steven Pavletic; Taiga Nishihori; Tim Prestidge; Vaibhav Agrawal; William J Hogan; Yoshihiro Inamoto; Bronwen E Shaw; Prakash Satwani
Journal:  Blood Adv       Date:  2020-05-12

8.  Normal IgH Repertoire Diversity in an Infant with ADA Deficiency After Gene Therapy.

Authors:  Carolyn H Baloh; Samiksha A Borkar; Kai-Fen Chang; Jiqiang Yao; Michael S Hershfield; Suhag H Parikh; Donald B Kohn; Maureen M Goodenow; John W Sleasman; Li Yin
Journal:  J Clin Immunol       Date:  2021-06-28       Impact factor: 8.317

9.  Expectations and experience: Parent and patient perspectives regarding treatment for Severe Combined Immunodeficiency (SCID).

Authors:  Heather Smith; Christopher Scalchunes; Morton J Cowan; Jennifer Puck; Jennifer Heimall
Journal:  Clin Immunol       Date:  2021-06-16       Impact factor: 10.190

10.  Asymptomatic Infant With Atypical SCID and Novel Hypomorphic RAG Variant Identified by Newborn Screening: A Diagnostic and Treatment Dilemma.

Authors:  Maria Chitty-Lopez; Emma Westermann-Clark; Irina Dawson; Boglarka Ujhazi; Krisztian Csomos; Kerry Dobbs; Khuong Le; Yasuhiro Yamazaki; Amir A Sadighi Akha; Deepak Chellapandian; Ben Oshrine; Luigi D Notarangelo; Gauri Sunkersett; Jennifer W Leiding; Jolan E Walter
Journal:  Front Immunol       Date:  2020-09-29       Impact factor: 7.561
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