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

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

doi:10.1182/bloodadvances.2019000839

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

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 DuncanEdward 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

Hematology

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Abstract

We examined the risk of subsequent neoplasms (SNs) and late mortality in children and adolescents undergoing allogeneic hematopoietic cell transplantation (HCT) for nonmalignant diseases (NMDs). We included 6028 patients (median age, 6 years; interquartile range, 1-11; range,,1 to 20) from the Center for International Blood and Marrow Transplant Research (1995-2012) registry. Standardized mortality ratios (SMRs) in 2-year survivors and standardized incidence ratios (SIRs) were calculated to compare mortality and SN rates with expected rates in the general population. Median follow-up of survivors was 7.8 years. Diagnoses included severe aplastic anemia (SAA; 24%), Fanconi anemia (FA; 10%), other marrow failure (6%), hemoglobinopathy (15%), immunodeficiency (23%), and metabolic/ leukodystrophy syndrome (22%). Ten-year survival was 93% (95% confidence interval [95% CI], 92% to 94%; SMR, 4.2; 95% CI, 3.7-4.8). Seventy-one patients developed SNs (1.2%). Incidence was highest in FA (5.5%), SAA (1.1%), and other marrow failure syndromes (1.7%); for other NMDs, incidence was,1%. Hematologic (27%), oropharyngeal (25%), and skin cancers (13%) were most common. Leukemia risk was highest in the first 5 years posttransplantation; oropharyngeal, skin, liver, and thyroid tumors primarily occurred after 5 years. Despite a low number of SNs, patients had an 11-fold increased SN risk (SIR, 11; 95% CI, 8.9-13.9) compared with the general population. We report excellent long-term survival and low SN incidence in an international cohort of children undergoing HCT for NMDs. The risk of SN development was highest in patients with FA and marrow failure syndromes, highlighting the need for long-term posttransplantation surveillance in this population.

Original language	English (US)
Pages (from-to)	2084-2094
Number of pages	11
Journal	Blood Advances
Volume	4
Issue number	9
DOIs	https://doi.org/10.1182/bloodadvances.2019000839
State	Published - May 12 2020

ASJC Scopus subject areas

Hematology

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10.1182/bloodadvances.2019000839

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Kahn, J. M., Brazauskas, R., Tecca, H. R., Bo-Subait, S., Buchbinder, D., Battiwala, M., Flowers, M. E. D., Savani, B. N., Phelan, R., Broglie, L., Abraham, A. A., Keating, A. K., Daly, A., Wirk, B., George, B., Alter, B. P., Ustun, C., Freytes, C. O., Beitinjaneh, A. M., ... Satwani, P. (2020). Subsequent neoplasms and late mortality in children undergoing allogeneic transplantation for nonmalignant diseases. Blood Advances, 4(9), 2084-2094. https://doi.org/10.1182/bloodadvances.2019000839

Kahn, JM, Brazauskas, R, Tecca, HR, Bo-Subait, S, Buchbinder, D, Battiwala, M, Flowers, MED, Savani, BN, Phelan, R, Broglie, L, Abraham, AA, Keating, AK, Daly, A, Wirk, B, George, B, Alter, BP, Ustun, C, Freytes, CO, Beitinjaneh, AM, Duncan, C, Copelan, E, Hildebrandt, GC, Murthy, HS, Lazarus, HM, Auletta, JJ, Myers, KC, Williams, KM, Page, KM, Vrooman, LM, Norkin, M, Byrne, M, Diaz, MA, Kamani, N, Bhatt, NS, Rezvani, A, Farhadfar, N, Mehta, PA, Hematti, P, Shaw, PJ, Kamble, RT, Schears, R, Olsson, RF, Hayashi, RJ, Gale, RP, Mayo, SJ, Chhabra, S, Rotz, SJ, Badawy, SM, Ganguly, S, Pavletic, S, Nishihori, T, Prestidge, T, Agrawal, V, Hogan, WJ, Inamoto, Y, Shaw, BE & Satwani, P 2020, 'Subsequent neoplasms and late mortality in children undergoing allogeneic transplantation for nonmalignant diseases', Blood Advances, vol. 4, no. 9, pp. 2084-2094. https://doi.org/10.1182/bloodadvances.2019000839

@article{37b973e95053423c8e48aec390bbe4c3,

title = "Subsequent neoplasms and late mortality in children undergoing allogeneic transplantation for nonmalignant diseases",

abstract = "We examined the risk of subsequent neoplasms (SNs) and late mortality in children and adolescents undergoing allogeneic hematopoietic cell transplantation (HCT) for nonmalignant diseases (NMDs). We included 6028 patients (median age, 6 years; interquartile range, 1-11; range,,1 to 20) from the Center for International Blood and Marrow Transplant Research (1995-2012) registry. Standardized mortality ratios (SMRs) in 2-year survivors and standardized incidence ratios (SIRs) were calculated to compare mortality and SN rates with expected rates in the general population. Median follow-up of survivors was 7.8 years. Diagnoses included severe aplastic anemia (SAA; 24%), Fanconi anemia (FA; 10%), other marrow failure (6%), hemoglobinopathy (15%), immunodeficiency (23%), and metabolic/ leukodystrophy syndrome (22%). Ten-year survival was 93% (95% confidence interval [95% CI], 92% to 94%; SMR, 4.2; 95% CI, 3.7-4.8). Seventy-one patients developed SNs (1.2%). Incidence was highest in FA (5.5%), SAA (1.1%), and other marrow failure syndromes (1.7%); for other NMDs, incidence was,1%. Hematologic (27%), oropharyngeal (25%), and skin cancers (13%) were most common. Leukemia risk was highest in the first 5 years posttransplantation; oropharyngeal, skin, liver, and thyroid tumors primarily occurred after 5 years. Despite a low number of SNs, patients had an 11-fold increased SN risk (SIR, 11; 95% CI, 8.9-13.9) compared with the general population. We report excellent long-term survival and low SN incidence in an international cohort of children undergoing HCT for NMDs. The risk of SN development was highest in patients with FA and marrow failure syndromes, highlighting the need for long-term posttransplantation surveillance in this population.",

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

year = "2020",

month = may,

day = "12",

doi = "10.1182/bloodadvances.2019000839",

language = "English (US)",

volume = "4",

pages = "2084--2094",

journal = "Blood Advances",

issn = "2473-9529",

publisher = "American Society of Hematology",

number = "9",

}

TY - JOUR

T1 - Subsequent neoplasms and late mortality in children undergoing allogeneic transplantation for nonmalignant diseases

AU - Kahn, Justine M.

AU - Brazauskas, Ruta

AU - Tecca, Heather R.

AU - Bo-Subait, Stephanie

AU - Buchbinder, David

AU - Battiwala, Minoo

AU - Flowers, Mary E.D.

AU - Savani, Bipin N.

AU - Phelan, Rachel

AU - Broglie, Larisa

AU - Abraham, Allistair A.

AU - Keating, Amy K.

AU - Daly, Andrew

AU - Wirk, Baldeep

AU - George, Biju

AU - Alter, Blanche P.

AU - Ustun, Celalettin

AU - Freytes, Cesar O.

AU - Beitinjaneh, Amer M.

AU - Duncan, Christine

AU - Copelan, Edward

AU - Hildebrandt, Gerhard C.

AU - Murthy, Hemant S.

AU - Lazarus, Hillard M.

AU - Auletta, Jeffery J.

AU - Myers, Kasiani C.

AU - Williams, Kirsten M.

AU - Page, Kristin M.

AU - Vrooman, Lynda M.

AU - Norkin, Maxim

AU - Byrne, Michael

AU - Diaz, Miguel Angel

AU - Kamani, Naynesh

AU - Bhatt, Neel S.

AU - Rezvani, Andrew

AU - Farhadfar, Nosha

AU - Mehta, Parinda A.

AU - Hematti, Peiman

AU - Shaw, Peter J.

AU - Kamble, Rammurti T.

AU - Schears, Raquel

AU - Olsson, Richard F.

AU - Hayashi, Robert J.

AU - Gale, Robert Peter

AU - Mayo, Samantha J.

AU - Chhabra, Saurabh

AU - Rotz, Seth J.

AU - Badawy, Sherif M.

AU - Ganguly, Siddhartha

AU - Pavletic, Steven

AU - Nishihori, Taiga

AU - Prestidge, Tim

AU - Agrawal, Vaibhav

AU - Hogan, William J.

AU - Inamoto, Yoshihiro

AU - Shaw, Bronwen E.

AU - Satwani, Prakash

PY - 2020/5/12

Y1 - 2020/5/12

N2 - We examined the risk of subsequent neoplasms (SNs) and late mortality in children and adolescents undergoing allogeneic hematopoietic cell transplantation (HCT) for nonmalignant diseases (NMDs). We included 6028 patients (median age, 6 years; interquartile range, 1-11; range,,1 to 20) from the Center for International Blood and Marrow Transplant Research (1995-2012) registry. Standardized mortality ratios (SMRs) in 2-year survivors and standardized incidence ratios (SIRs) were calculated to compare mortality and SN rates with expected rates in the general population. Median follow-up of survivors was 7.8 years. Diagnoses included severe aplastic anemia (SAA; 24%), Fanconi anemia (FA; 10%), other marrow failure (6%), hemoglobinopathy (15%), immunodeficiency (23%), and metabolic/ leukodystrophy syndrome (22%). Ten-year survival was 93% (95% confidence interval [95% CI], 92% to 94%; SMR, 4.2; 95% CI, 3.7-4.8). Seventy-one patients developed SNs (1.2%). Incidence was highest in FA (5.5%), SAA (1.1%), and other marrow failure syndromes (1.7%); for other NMDs, incidence was,1%. Hematologic (27%), oropharyngeal (25%), and skin cancers (13%) were most common. Leukemia risk was highest in the first 5 years posttransplantation; oropharyngeal, skin, liver, and thyroid tumors primarily occurred after 5 years. Despite a low number of SNs, patients had an 11-fold increased SN risk (SIR, 11; 95% CI, 8.9-13.9) compared with the general population. We report excellent long-term survival and low SN incidence in an international cohort of children undergoing HCT for NMDs. The risk of SN development was highest in patients with FA and marrow failure syndromes, highlighting the need for long-term posttransplantation surveillance in this population.

AB - We examined the risk of subsequent neoplasms (SNs) and late mortality in children and adolescents undergoing allogeneic hematopoietic cell transplantation (HCT) for nonmalignant diseases (NMDs). We included 6028 patients (median age, 6 years; interquartile range, 1-11; range,,1 to 20) from the Center for International Blood and Marrow Transplant Research (1995-2012) registry. Standardized mortality ratios (SMRs) in 2-year survivors and standardized incidence ratios (SIRs) were calculated to compare mortality and SN rates with expected rates in the general population. Median follow-up of survivors was 7.8 years. Diagnoses included severe aplastic anemia (SAA; 24%), Fanconi anemia (FA; 10%), other marrow failure (6%), hemoglobinopathy (15%), immunodeficiency (23%), and metabolic/ leukodystrophy syndrome (22%). Ten-year survival was 93% (95% confidence interval [95% CI], 92% to 94%; SMR, 4.2; 95% CI, 3.7-4.8). Seventy-one patients developed SNs (1.2%). Incidence was highest in FA (5.5%), SAA (1.1%), and other marrow failure syndromes (1.7%); for other NMDs, incidence was,1%. Hematologic (27%), oropharyngeal (25%), and skin cancers (13%) were most common. Leukemia risk was highest in the first 5 years posttransplantation; oropharyngeal, skin, liver, and thyroid tumors primarily occurred after 5 years. Despite a low number of SNs, patients had an 11-fold increased SN risk (SIR, 11; 95% CI, 8.9-13.9) compared with the general population. We report excellent long-term survival and low SN incidence in an international cohort of children undergoing HCT for NMDs. The risk of SN development was highest in patients with FA and marrow failure syndromes, highlighting the need for long-term posttransplantation surveillance in this population.

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JO - Blood Advances

JF - Blood Advances

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ER -

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

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