Physician decision making in selection of second-line treatments in immune thrombocytopenia in children

Rachael F. Grace, Jenny M. Despotovic, Carolyn M. Bennett, James B. Bussel, Michelle Neier, Cindy Neunert, Shelley E. Crary, Yves D. Pastore, Robert J. Klaassen, Jennifer A. Rothman, Kerry Hege, Vicky R. Breakey, Melissa J. Rose, Kristin A. Shimano, George R. Buchanan, Amy Geddis, Kristina M. Haley, Adonis Lorenzana, Alexis Thompson, Michael Jeng & 4 others Ellis J. Neufeld, Travis Brown, Peter W. Forbes, Michele P. Lambert

Research output: Contribution to journalArticle

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Abstract

Immune thrombocytopenia (ITP) is an acquired autoimmune bleeding disorder which presents with isolated thrombocytopenia and risk of hemorrhage. While most children with ITP promptly recover with or without drug therapy, ITP is persistent or chronic in others. When needed, how to select second-line therapies is not clear. ICON1, conducted within the Pediatric ITP Consortium of North America (ICON), is a prospective, observational, longitudinal cohort study of 120 children from 21 centers starting second-line treatments for ITP which examined treatment decisions. Treating physicians reported reasons for selecting therapies, ranking the top three. In a propensity weighted model, the most important factors were patient/parental preference (53%) and treatment-related factors: side effect profile (58%), long-term toxicity (54%), ease of administration (46%), possibility of remission (45%), and perceived efficacy (30%). Physician, health system, and clinical factors rarely influenced decision-making. Patient/parent preferences were selected as reasons more often in chronic ITP (85.7%) than in newly diagnosed (0%) or persistent ITP (14.3%, P =.003). Splenectomy and rituximab were chosen for the possibility of inducing long-term remission (P <.001). Oral agents, such as eltrombopag and immunosuppressants, were chosen for ease of administration and expected adherence (P <.001). Physicians chose rituximab in patients with lower expected adherence (P =.017). Treatment choice showed some physician and treatment center bias. This study illustrates the complexity and many factors involved in decision-making in selecting second-line ITP treatments, given the absence of comparative trials. It highlights shared decision-making and the need for well-conducted, comparative effectiveness studies to allow for informed discussion between patients and clinicians.

Original languageEnglish (US)
Pages (from-to)882-888
Number of pages7
JournalAmerican Journal of Hematology
Volume93
Issue number7
DOIs
StatePublished - Jul 1 2018

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Idiopathic Thrombocytopenic Purpura
Decision Making
Physicians
Patient Preference
Therapeutics
Hemorrhage
Splenectomy
Immunosuppressive Agents
North America
Thrombocytopenia
Longitudinal Studies
Cohort Studies
Pediatrics
Drug Therapy
Health

All Science Journal Classification (ASJC) codes

  • Hematology

Cite this

Grace, R. F., Despotovic, J. M., Bennett, C. M., Bussel, J. B., Neier, M., Neunert, C., ... Lambert, M. P. (2018). Physician decision making in selection of second-line treatments in immune thrombocytopenia in children. American Journal of Hematology, 93(7), 882-888. https://doi.org/10.1002/ajh.25110
Grace, Rachael F. ; Despotovic, Jenny M. ; Bennett, Carolyn M. ; Bussel, James B. ; Neier, Michelle ; Neunert, Cindy ; Crary, Shelley E. ; Pastore, Yves D. ; Klaassen, Robert J. ; Rothman, Jennifer A. ; Hege, Kerry ; Breakey, Vicky R. ; Rose, Melissa J. ; Shimano, Kristin A. ; Buchanan, George R. ; Geddis, Amy ; Haley, Kristina M. ; Lorenzana, Adonis ; Thompson, Alexis ; Jeng, Michael ; Neufeld, Ellis J. ; Brown, Travis ; Forbes, Peter W. ; Lambert, Michele P. / Physician decision making in selection of second-line treatments in immune thrombocytopenia in children. In: American Journal of Hematology. 2018 ; Vol. 93, No. 7. pp. 882-888.
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abstract = "Immune thrombocytopenia (ITP) is an acquired autoimmune bleeding disorder which presents with isolated thrombocytopenia and risk of hemorrhage. While most children with ITP promptly recover with or without drug therapy, ITP is persistent or chronic in others. When needed, how to select second-line therapies is not clear. ICON1, conducted within the Pediatric ITP Consortium of North America (ICON), is a prospective, observational, longitudinal cohort study of 120 children from 21 centers starting second-line treatments for ITP which examined treatment decisions. Treating physicians reported reasons for selecting therapies, ranking the top three. In a propensity weighted model, the most important factors were patient/parental preference (53{\%}) and treatment-related factors: side effect profile (58{\%}), long-term toxicity (54{\%}), ease of administration (46{\%}), possibility of remission (45{\%}), and perceived efficacy (30{\%}). Physician, health system, and clinical factors rarely influenced decision-making. Patient/parent preferences were selected as reasons more often in chronic ITP (85.7{\%}) than in newly diagnosed (0{\%}) or persistent ITP (14.3{\%}, P =.003). Splenectomy and rituximab were chosen for the possibility of inducing long-term remission (P <.001). Oral agents, such as eltrombopag and immunosuppressants, were chosen for ease of administration and expected adherence (P <.001). Physicians chose rituximab in patients with lower expected adherence (P =.017). Treatment choice showed some physician and treatment center bias. This study illustrates the complexity and many factors involved in decision-making in selecting second-line ITP treatments, given the absence of comparative trials. It highlights shared decision-making and the need for well-conducted, comparative effectiveness studies to allow for informed discussion between patients and clinicians.",
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Grace, RF, Despotovic, JM, Bennett, CM, Bussel, JB, Neier, M, Neunert, C, Crary, SE, Pastore, YD, Klaassen, RJ, Rothman, JA, Hege, K, Breakey, VR, Rose, MJ, Shimano, KA, Buchanan, GR, Geddis, A, Haley, KM, Lorenzana, A, Thompson, A, Jeng, M, Neufeld, EJ, Brown, T, Forbes, PW & Lambert, MP 2018, 'Physician decision making in selection of second-line treatments in immune thrombocytopenia in children', American Journal of Hematology, vol. 93, no. 7, pp. 882-888. https://doi.org/10.1002/ajh.25110

Physician decision making in selection of second-line treatments in immune thrombocytopenia in children. / Grace, Rachael F.; Despotovic, Jenny M.; Bennett, Carolyn M.; Bussel, James B.; Neier, Michelle; Neunert, Cindy; Crary, Shelley E.; Pastore, Yves D.; Klaassen, Robert J.; Rothman, Jennifer A.; Hege, Kerry; Breakey, Vicky R.; Rose, Melissa J.; Shimano, Kristin A.; Buchanan, George R.; Geddis, Amy; Haley, Kristina M.; Lorenzana, Adonis; Thompson, Alexis; Jeng, Michael; Neufeld, Ellis J.; Brown, Travis; Forbes, Peter W.; Lambert, Michele P.

In: American Journal of Hematology, Vol. 93, No. 7, 01.07.2018, p. 882-888.

Research output: Contribution to journalArticle

TY - JOUR

T1 - Physician decision making in selection of second-line treatments in immune thrombocytopenia in children

AU - Grace, Rachael F.

AU - Despotovic, Jenny M.

AU - Bennett, Carolyn M.

AU - Bussel, James B.

AU - Neier, Michelle

AU - Neunert, Cindy

AU - Crary, Shelley E.

AU - Pastore, Yves D.

AU - Klaassen, Robert J.

AU - Rothman, Jennifer A.

AU - Hege, Kerry

AU - Breakey, Vicky R.

AU - Rose, Melissa J.

AU - Shimano, Kristin A.

AU - Buchanan, George R.

AU - Geddis, Amy

AU - Haley, Kristina M.

AU - Lorenzana, Adonis

AU - Thompson, Alexis

AU - Jeng, Michael

AU - Neufeld, Ellis J.

AU - Brown, Travis

AU - Forbes, Peter W.

AU - Lambert, Michele P.

PY - 2018/7/1

Y1 - 2018/7/1

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