Management of Children with newly Diagnosed ITP: the Toronto - - PowerPoint PPT Presentation
Shared Decision Making in the Management of Children with newly Diagnosed ITP: the Toronto Experience Dr Victor Blanchette Professor of Pediatrics University of Toronto Medical Director, Pediatric Thrombosis and Hemostasis Program Division of
Professor of Pediatrics University of Toronto Medical Director, Pediatric Thrombosis and Hemostasis Program Division of Hematology/Oncology The Hospital for Sick Children Toronto, Canada
Newly Diagnosed ITP ITP during the first 3 months of the illness replaces term acute ITP Persistent ITP ITP lasting between 3 to 12 months Chronic ITP ITP lasting for > 12 months Platelet “threshold” for definition of ITP < 100 x 109 /L
Rodeghiero F et al Blood 2009 ;113 :2386-2393
The typical child with newly diagnosed ITP is:
thrombocytopenia and has
petechial rash
BLOOD SMEAR SHOWING A MEGATHROMBOCYTE BONE MARROW ASPIRATE SHOWING INCREASED NUMBERS OF MEGAKARYOCYTES
None or mild (76%)3 no bleeding at all or bruising, petechiae, occasional mild epistaxis with little or no interference with daily living (“dry purpura”) Moderate (21%) more severe skin manifestations with some mucosal lesions and more tolerable epistaxis and menorrhagia (“wet purpura”) Severe (3%) bleeding episodes (epistaxis, melena, menorrhagia, and/or intracranial hemorrhage) requiring hospitalization and/or blood transfusions
(1) Bolton-Maggs PH et al Lancet 1997;350:620-623 (2) Bolton-Maggs PH et al J Pediatr Hematol Oncol 2003;25(Suppl 1):S47-S51 (3) Neunert C et al Blood 2008;112:4003 - 4008
N = 863 (2001-2004)
Risk factors for intracranial hemorrhage (ICH) in children with acute ITP :
Incidence of ICH = approx. 2/500 cases ( 0.4% )
Neunert C et al J Thromb Haemost 2015;13:457-465
Percent (%) of children with platelet count > 20 x 109/L at 72 hours following start of management strategy IVIG 0.8 g/kg x 1* 97% (34/35) IVIG 1.0g/kg x 2 94% (50/53) IV anti-D 82% (31/38) Oral prednisone** 79% (45/57) Observation (“no therapy”) 56% (9/16)
Blanchette V et al J Pediatr 1993;123:989-995 Blanchette V et al Lancet 1994;344:703-707 *IVIG = Intravenous Immunoglobulin G **4mg/kg/day in divided doses (maximum dose = 150 mg/day)
Acta Paediatrica Scandinavica 424 : 71 - 74 ; 1998
Prednisone 4 mg/kg/ day x 4 days without tapering N = 25 Age 0.3 - 16 yr ( mean 6.4 ) Platelet count 1 - 14 x 109 / L ( mean 5 )
8% 42% 65%
Calpin C Arch Pediatric Adolescent Med 1998;152:345-347
“ the majority of children with newly diagnosed ITP lack significant bleeding symptoms and may be managed without therapy directed at raising the platelet count at the discretion of the hematologist and the patient” and “it is necessary to treat all children with severe bleeding symptoms and treatment should also be considered in children with moderate bleeding or those at increased risk of bleeding”
Provan D et al Blood 2010;115:168-186 (online publication October 21, 2009)
“ children with no bleeding or mild bleeding (defined as skin manifestations only such as bruising and petechiae) be managed with
and “for pediatric patients requiring treatment a single dose of IVIG (0.8 to 1 g/kg) or a short course of corticosteroids be used as first-line treatment”
Neunert C et al blood 2011;117(16):4190-4207 (first published on line February, 2011)
ISTH Definitions in Hemophilia Project Group : Personal communication V. Blanchette (2013)
Management Option UK (1) (2007 - 2009)* Intercontinental ITP Study Group(2) Hospital for Sick Children Toronto (2007-2009)(3) Observation IVIG Corticosteroids 84% 31% 29% 33% 6.3% 87.5% 6.3%
(1) Grainger JD et al Arch Dis Child 2012;97:8-11 (2) Kuhne T et al Lancet 2001;358-2122-2125 (3) Beck C Personal Communication
16%
*data extracted from UK Childhood ITP Registry
and their health care providers as they consider 2 or more clinically reasonable options
evidence related to each option including potential benefits, risks and alternatives to the options presented
Epstein RM et al JAMA 2009;3002:195-197 Charles C et al Soc Sci Med 1999; 49:651-661
GOAL OF SDM: identification of which course of action is most consistent with the preference of the patient and/or their parents/guardians
Participants Number of Comment Participants
Children with newly Median age 11 years diagnosed ITP* 7 (range 10-18 years) Parents of children Median age at diagnosis with newly diagnosed 3.25 years
6 Parents of children with Median age at diagnosis chronic ITP 11 7.25 years Health care professionals 10 10 MD’s (4 trainees); 1 nurse
* Onset of ITP ≤ 2 years before participation in the focus group
Beck C. et al J Pediatr Hematol Oncol 2014;36:559-565
Beck CE J Pediatr Hematol Oncol 2014;36(7):559-565
Treatment choice “so many things (were) running through my head” Re explanation from doctors: “really big words”; “scarry stuff” “… they basically said… this is what we do” Steroids were discussed as a treatment option but my parents said “no way”
Quotations Decision-making Themes Anxiety, fear, confusion Understanding information
Decision-making Themes Quotations Comfort level Assumptions Pending information Treatment choice “I would not take the risk, I would treat” “Nobody wants steroids” “It depends on how you say it. I can convince anybody to go on steroids” “The decision to treat with IVIG is easy but things need to change”
Child with newly diagnosed ITP
Child and/or parents/guardians given ITP information sheet and evidence based protocol outlining 3 management options
Meeting between child and/or parents/guardians and a member
staff to discuss management options
Beck CE, Personal Communication 2017
Blood Cell and Components Petechiae and Bruises
* www.aboutkidshealth.ca
Patients with Newly Diagnosed, Typical ITP : 3 months - 18 years
via Emergency Department or community hospital
Assessment
Treatment
Discharge
care hematology nurse
hematology clinic
www.aboutkidshealth.ca/EN/HealthAZ/ConditionsandDiseases/blooddisorders/Pages/ITP-what-happens-after-diagnosis.aspx
Study cohort
Source Material*
2008 - 2010(1) 2008 - 2014(2) Number of cases 2,314 4,937 Management % IVIG 72.2 68.8 Steroids 7.8 4.5 Anti-D 4.8 5.6 IVIG and anti-D 5.8 ND Combination ND 14.5 Observation 9.4 6.6
*Pediatric Health Information System database
(1) Kime C et al Pediatrics 2013;131:880-885 (2) Witmer C et al Pediatr Blood Cancer 2016;63:1227-1231
Schultz CL et al 2014;JAMA Pediatrics;168(10):1-7
Klaassen RJ et al Pediatr Blood Cancer 2013;60:95-100