Patient characteristics and outcomes in adolescents and young adults with classical Philadelphia chromosome-negative myeloproliferative neoplasms

Prajwal Boddu, Lucia Masarova, Srdan Verstovsek, Paolo Strati, Hagop Kantarjian, Jorge Cortes, Zeev Estrov, Sherry Pierce, Naveen Pemmaraju

Research output: Contribution to journalArticle

Abstract

Little is known about the outcomes of Philadelphia-negative myeloproliferative neoplasms (MPNs) in adolescents and young adults (AYA). We reviewed all patients with essential thrombocythemia (ET), polycythemia vera (PV), and myelofibrosis (MF) treated at our institution from 1988 to 2016 who were aged 16 to 39 years (AYA) and described their outcomes in comparison to older MPN population. Of 2206 patients, 185 (8.3%) were identified as AYA: 105 (57%) ET, 43 (23%) PV, and 37 (20%) MF. The median age was 33 years [range, 16–39], and median follow-up time 3 years [range, 0.04–25]. JAK2 allele burdens were significantly lower among AYA JAK2V617F-mutated patients in both PV (p = 0.001) and MF (p = 0.005). Seven percent of MPN AYA patients were diagnosed with a thrombotic event at, or prior to, diagnosis. Over the short median follow-up, 4 thrombotic (PV = 1, MF = 3) and 3 leukemia (ET = 2, MF = 1) events occurred. In multivariate analysis, AYA did not predict for thrombotic or transformational events across three cohorts. In the MF cohort, there was a reduced frequency of negative prognostic variables of anemia (p = 0.011) and leukocytosis (p = 0.048) in AYA when compared with non-AYA. Overall survival was significantly superior in the AYA cohorts in all three MPN groups, namely MF (p < 0.001), PV (p < 0.001), and ET (p = 0.002). Our findings suggest that MPN AYA patients exhibit an indolent clinical phenotype characterized by favorable survival outcomes.

Original languageEnglish (US)
Pages (from-to)109-121
Number of pages13
JournalAnnals of Hematology
Volume97
Issue number1
DOIs
StatePublished - Jan 1 2018
Externally publishedYes

Fingerprint

Philadelphia Chromosome
Primary Myelofibrosis
Young Adult
Polycythemia Vera
Essential Thrombocythemia
Neoplasms
Survival
Leukocytosis
Anemia
Leukemia
Multivariate Analysis
Alleles
Phenotype

Keywords

  • Adolescent
  • Essential thrombocythemia
  • Myelofibrosis
  • Myeloproliferative neoplasm
  • Polycythemia vera
  • Young adults

ASJC Scopus subject areas

  • Hematology

Cite this

Patient characteristics and outcomes in adolescents and young adults with classical Philadelphia chromosome-negative myeloproliferative neoplasms. / Boddu, Prajwal; Masarova, Lucia; Verstovsek, Srdan; Strati, Paolo; Kantarjian, Hagop; Cortes, Jorge; Estrov, Zeev; Pierce, Sherry; Pemmaraju, Naveen.

In: Annals of Hematology, Vol. 97, No. 1, 01.01.2018, p. 109-121.

Research output: Contribution to journalArticle

Boddu, Prajwal ; Masarova, Lucia ; Verstovsek, Srdan ; Strati, Paolo ; Kantarjian, Hagop ; Cortes, Jorge ; Estrov, Zeev ; Pierce, Sherry ; Pemmaraju, Naveen. / Patient characteristics and outcomes in adolescents and young adults with classical Philadelphia chromosome-negative myeloproliferative neoplasms. In: Annals of Hematology. 2018 ; Vol. 97, No. 1. pp. 109-121.
@article{edbde093408741dca9afa37c2cf065ec,
title = "Patient characteristics and outcomes in adolescents and young adults with classical Philadelphia chromosome-negative myeloproliferative neoplasms",
abstract = "Little is known about the outcomes of Philadelphia-negative myeloproliferative neoplasms (MPNs) in adolescents and young adults (AYA). We reviewed all patients with essential thrombocythemia (ET), polycythemia vera (PV), and myelofibrosis (MF) treated at our institution from 1988 to 2016 who were aged 16 to 39 years (AYA) and described their outcomes in comparison to older MPN population. Of 2206 patients, 185 (8.3{\%}) were identified as AYA: 105 (57{\%}) ET, 43 (23{\%}) PV, and 37 (20{\%}) MF. The median age was 33 years [range, 16–39], and median follow-up time 3 years [range, 0.04–25]. JAK2 allele burdens were significantly lower among AYA JAK2V617F-mutated patients in both PV (p = 0.001) and MF (p = 0.005). Seven percent of MPN AYA patients were diagnosed with a thrombotic event at, or prior to, diagnosis. Over the short median follow-up, 4 thrombotic (PV = 1, MF = 3) and 3 leukemia (ET = 2, MF = 1) events occurred. In multivariate analysis, AYA did not predict for thrombotic or transformational events across three cohorts. In the MF cohort, there was a reduced frequency of negative prognostic variables of anemia (p = 0.011) and leukocytosis (p = 0.048) in AYA when compared with non-AYA. Overall survival was significantly superior in the AYA cohorts in all three MPN groups, namely MF (p < 0.001), PV (p < 0.001), and ET (p = 0.002). Our findings suggest that MPN AYA patients exhibit an indolent clinical phenotype characterized by favorable survival outcomes.",
keywords = "Adolescent, Essential thrombocythemia, Myelofibrosis, Myeloproliferative neoplasm, Polycythemia vera, Young adults",
author = "Prajwal Boddu and Lucia Masarova and Srdan Verstovsek and Paolo Strati and Hagop Kantarjian and Jorge Cortes and Zeev Estrov and Sherry Pierce and Naveen Pemmaraju",
year = "2018",
month = "1",
day = "1",
doi = "10.1007/s00277-017-3165-9",
language = "English (US)",
volume = "97",
pages = "109--121",
journal = "Annals of Hematology",
issn = "0939-5555",
publisher = "Springer Verlag",
number = "1",

}

TY - JOUR

T1 - Patient characteristics and outcomes in adolescents and young adults with classical Philadelphia chromosome-negative myeloproliferative neoplasms

AU - Boddu, Prajwal

AU - Masarova, Lucia

AU - Verstovsek, Srdan

AU - Strati, Paolo

AU - Kantarjian, Hagop

AU - Cortes, Jorge

AU - Estrov, Zeev

AU - Pierce, Sherry

AU - Pemmaraju, Naveen

PY - 2018/1/1

Y1 - 2018/1/1

N2 - Little is known about the outcomes of Philadelphia-negative myeloproliferative neoplasms (MPNs) in adolescents and young adults (AYA). We reviewed all patients with essential thrombocythemia (ET), polycythemia vera (PV), and myelofibrosis (MF) treated at our institution from 1988 to 2016 who were aged 16 to 39 years (AYA) and described their outcomes in comparison to older MPN population. Of 2206 patients, 185 (8.3%) were identified as AYA: 105 (57%) ET, 43 (23%) PV, and 37 (20%) MF. The median age was 33 years [range, 16–39], and median follow-up time 3 years [range, 0.04–25]. JAK2 allele burdens were significantly lower among AYA JAK2V617F-mutated patients in both PV (p = 0.001) and MF (p = 0.005). Seven percent of MPN AYA patients were diagnosed with a thrombotic event at, or prior to, diagnosis. Over the short median follow-up, 4 thrombotic (PV = 1, MF = 3) and 3 leukemia (ET = 2, MF = 1) events occurred. In multivariate analysis, AYA did not predict for thrombotic or transformational events across three cohorts. In the MF cohort, there was a reduced frequency of negative prognostic variables of anemia (p = 0.011) and leukocytosis (p = 0.048) in AYA when compared with non-AYA. Overall survival was significantly superior in the AYA cohorts in all three MPN groups, namely MF (p < 0.001), PV (p < 0.001), and ET (p = 0.002). Our findings suggest that MPN AYA patients exhibit an indolent clinical phenotype characterized by favorable survival outcomes.

AB - Little is known about the outcomes of Philadelphia-negative myeloproliferative neoplasms (MPNs) in adolescents and young adults (AYA). We reviewed all patients with essential thrombocythemia (ET), polycythemia vera (PV), and myelofibrosis (MF) treated at our institution from 1988 to 2016 who were aged 16 to 39 years (AYA) and described their outcomes in comparison to older MPN population. Of 2206 patients, 185 (8.3%) were identified as AYA: 105 (57%) ET, 43 (23%) PV, and 37 (20%) MF. The median age was 33 years [range, 16–39], and median follow-up time 3 years [range, 0.04–25]. JAK2 allele burdens were significantly lower among AYA JAK2V617F-mutated patients in both PV (p = 0.001) and MF (p = 0.005). Seven percent of MPN AYA patients were diagnosed with a thrombotic event at, or prior to, diagnosis. Over the short median follow-up, 4 thrombotic (PV = 1, MF = 3) and 3 leukemia (ET = 2, MF = 1) events occurred. In multivariate analysis, AYA did not predict for thrombotic or transformational events across three cohorts. In the MF cohort, there was a reduced frequency of negative prognostic variables of anemia (p = 0.011) and leukocytosis (p = 0.048) in AYA when compared with non-AYA. Overall survival was significantly superior in the AYA cohorts in all three MPN groups, namely MF (p < 0.001), PV (p < 0.001), and ET (p = 0.002). Our findings suggest that MPN AYA patients exhibit an indolent clinical phenotype characterized by favorable survival outcomes.

KW - Adolescent

KW - Essential thrombocythemia

KW - Myelofibrosis

KW - Myeloproliferative neoplasm

KW - Polycythemia vera

KW - Young adults

UR - http://www.scopus.com/inward/record.url?scp=85034066664&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=85034066664&partnerID=8YFLogxK

U2 - 10.1007/s00277-017-3165-9

DO - 10.1007/s00277-017-3165-9

M3 - Article

C2 - 29143068

AN - SCOPUS:85034066664

VL - 97

SP - 109

EP - 121

JO - Annals of Hematology

JF - Annals of Hematology

SN - 0939-5555

IS - 1

ER -