Type I and Ir pleuropulmonary blastoma (PPB): A report from the International PPB/DICER1 Registry

被引:13
|
作者
Nelson, Alexander T. [1 ,2 ,3 ,4 ]
Harris, Anne K. [1 ,2 ,3 ]
Watson, Dave [5 ]
Miniati, Doug [6 ]
Finch, Mike [5 ]
Kamihara, Junne [7 ]
Mitchell, Sarah G. [8 ]
Wilson, David B. [9 ]
Gettinger, Katie [9 ]
Rangaswami, Arun A. [10 ]
Campos, Jose M. [11 ]
Lederman, Sara [1 ,2 ,3 ,4 ]
Feltis, Brad A. [12 ]
Vasta, Lauren M. [13 ]
Harney, Laura A. [14 ]
Stewart, Douglas R. [13 ]
Dehner, Louis P. [15 ]
Messinger, Yoav H. [1 ,2 ,3 ]
Hill, D. Ashley [16 ,17 ]
Schultz, Kris Ann P. [1 ,2 ,3 ]
机构
[1] Childrens Minnesota, Int Pleuropulm Blastoma DICER1 Registry, Minneapolis, MN USA
[2] Childrens Minnesota, Int Ovarian & Testicular Stromal Tumor Registry, Minneapolis, MN USA
[3] Childrens Minnesota, Canc & Blood Disorders, Minneapolis, MN USA
[4] Univ Minnesota, Med Sch, Minneapolis, MN USA
[5] Childrens Minnesota, Res & Sponsored Programs, Minneapolis, MN USA
[6] Kaiser Permanente Northern Calif, Div Pediat Surg, Roseville, CA USA
[7] Boston Childrens Hosp, Dana Farber Canc Inst, Pediat Oncol, Boston, MA USA
[8] Emory Univ, Sch Med, Childrens Healthcare Atlanta, Aflac Canc & Blood Disorders Ctr, Atlanta, GA USA
[9] Washington Univ, Sch Med, Dept Pediat, St Louis Childrens Hosp, St Louis, MO 63110 USA
[10] Univ Calif San Francisco, Div Pediat Hematol & Oncol, San Francisco, CA 94143 USA
[11] Hosp Dr Sotero del Rio, Div Pediat Surg, Santiago, Chile
[12] East Tennessee State Univ, Div Pediat Surg, Johnson City, TN USA
[13] NCI, Clin Genet Branch, Div Canc Epidemiol & Genet, Rockville, MD USA
[14] Westat Corp, Rockville, MD USA
[15] Washington Univ, Med Ctr, Dept Pathol & Immunol, Lauren V Ackerman Lab Surg Pathol, St Louis, MO USA
[16] Childrens Natl Med Ctr, Dept Pathol & Lab Med, Washington, DC 20010 USA
[17] ResourcePath LLC, Sterling, VA USA
基金
美国国家卫生研究院;
关键词
DICER1; DICER1 tumor predisposition; lung cyst; pleuropulmonary blastoma; type I PPB; type Ir PPB; SOMATIC DICER1 MUTATIONS; GERM-LINE; OVARIAN; PATHOLOGY; TUMOR;
D O I
10.1002/cncr.34593
中图分类号
R73 [肿瘤学];
学科分类号
100214 ;
摘要
BackgroundPleuropulmonary blastoma (PPB) is the most common lung cancer of infancy and early childhood. Type I PPB is a purely cystic lesion that has a microscopic population of primitive small cells with or without rhabdomyoblastic features and may progress to type II or III PPB, whereas type Ir lacks primitive small cells. MethodsChildren with suspected PPB were enrolled in the International PPB/DICER1 Registry. Pathology was centrally reviewed, and follow-up was ascertained annually. ResultsBetween 2006 and 2022, 205 children had centrally reviewed type I or Ir PPB; 39% of children with type I and 5% of children with type Ir PPB received chemotherapy. Outcomes were favorable, although 11 children (nine with type I and two with type Ir PPB) experienced progression to type II/III (n = 8) or regrowth of type I PPB at the surgical site (n = 3), none of whom received chemotherapy before progression. Age and cyst size in combination were more suitable than either factor alone in predicting whether a particular lesion was type I or Ir PPB. ConclusionsFor young children with type I PPB, outcomes are favorable, but complete resection is indicated because of the risk for progression. Chemotherapy may be useful in a subset of children at increased risk for recurrence/progression. Efforts to risk stratify children with type I PPB to optimize outcomes while reducing treatment-related side effects are underway.
引用
收藏
页码:600 / 613
页数:14
相关论文
共 40 条
  • [31] Pleuropulmonary blastoma-like peritoneal sarcoma: a newly described malignancy associated with biallelic DICER1 pathogenic variation
    Schultz, Kris Ann P.
    Nelson, Alexander
    Harris, Anne K.
    Finch, Mike
    Field, Amanda
    Jarzembowski, Jason A.
    Wilhelm, Mercedes
    Mize, William
    Kreiger, Portia
    Conard, Katrina
    Walter, Andrew
    Olson, Thomas
    Mitchell, Sarah
    Runco, Daniel, V
    Bechtel, Allison
    Klawinski, Darren
    Bradfield, Scott
    Gettinger, Katie
    Stewart, Douglas R.
    Messinger, Yoav
    Dehner, Louis P.
    Hill, D. Ashley
    MODERN PATHOLOGY, 2020, 33 (10) : 1922 - 1929
  • [32] Cell-and developmental stage-specific Dicer1 ablation in the lung epithelium models cystic pleuropulmonary blastoma
    Wagh, Purnima K.
    Gardner, Margaret A.
    Ma, Xiaolan
    Callahan, Melissa
    Shannon, John M.
    Wert, Susan E.
    Messinger, Yoav H.
    Dehner, Louis P.
    Hill, D. Ashley
    Wikenheiser-Brokamp, Kathryn A.
    JOURNAL OF PATHOLOGY, 2015, 236 (01): : 41 - 52
  • [33] Nasal chondromesenchymal hamartomas arise secondary to germline and somatic mutations of DICER1 in the pleuropulmonary blastoma tumor predisposition disorder
    Douglas R. Stewart
    Yoav Messinger
    Gretchen M. Williams
    Jiandong Yang
    Amanda Field
    Kris Ann P. Schultz
    Laura A. Harney
    Leslie A. Doros
    Louis P. Dehner
    D. Ashley Hill
    Human Genetics, 2014, 133 : 1443 - 1450
  • [34] Ovarian tumors related to intronic mutations in DICER1: a report from the international ovarian and testicular stromal tumor registry
    Schultz, Kris Ann P.
    Harris, Anne
    Messinger, Yoav
    Sencer, Susan
    Baldinger, Shari
    Dehner, Louis P.
    Hill, D. Ashley
    FAMILIAL CANCER, 2016, 15 (01) : 105 - 110
  • [35] Type I pleuropulmonary blastoma presenting as congenital pulmonary airway malformation: A report of two cases
    Kumari, Kalpana
    Longchar, Moanaro
    Gunathilaka, Ganganath
    Narange, Priyanka
    Aggarwal, Sandeep
    Arava, Sudheer
    INDIAN JOURNAL OF PATHOLOGY AND MICROBIOLOGY, 2019, 62 (04) : 595 - 598
  • [36] Case report of a 3-year-old girl with pleuropulmonary blastoma and family history of a tumor predisposition syndrome with c. 2830 gene mutation in DICER1
    Puckett, Y.
    Howe, J.
    Vane, D.
    Agarwal, A.
    Batanian, J. R.
    Greenspon, J.
    JOURNAL OF PEDIATRIC SURGERY CASE REPORTS, 2015, 3 (08) : 312 - 315
  • [37] Pleuropulmonary blastoma type I presenting as a tension pneumothorax: a case report and review of the literature
    AbuAyyash, Rafat Ibrahim
    Aqel, Wafa
    Bannoura, Sami
    Asbeh, Yousef Abu
    ANNALS OF MEDICINE AND SURGERY, 2023, 85 (05): : 2024 - 2028
  • [38] Can congenital pulmonary airway malformation be distinguished from Type I pleuropulmonary blastoma based on clinical and radiological features?
    Feinberg, Adina
    Hall, Nigel J.
    Williams, Gretchen M.
    Schultz, Kris Ann P.
    Miniati, Doug
    Hill, D. Ashley
    Dehner, Louis P.
    Messinger, Yoav H.
    Langer, Jacob C.
    JOURNAL OF PEDIATRIC SURGERY, 2016, 51 (01) : 33 - 37
  • [39] Surveillance recommendations for DICER1 pathogenic variant carriers: a report from the SIOPE Host Genome Working Group and CanGene-CanVar Clinical Guideline Working Group
    Bakhuizen, Jette J.
    Hanson, Helen
    van der Tuin, Karin
    Lalloo, Fiona
    Tischkowitz, Marc
    Wadt, Karin
    Jongmans, Marjolijn C. J.
    FAMILIAL CANCER, 2021, 20 (04) : 337 - 348
  • [40] DICER1-related Sertoli-Leydig cell tumor and gynandroblastoma: Clinical and genetic findings from the International Ovarian and Testicular Stromal Tumor Registry
    Schultz, Kris Ann P.
    Harris, Anne K.
    Finch, Michael
    Dehner, Louis P.
    Brown, Jubilee B.
    Gershenson, David M.
    Young, Robert H.
    Field, Amanda
    Yu, Weiying
    Turner, Joyce
    Cost, Nicholas G.
    Schneider, Dominik T.
    Stewart, Douglas R.
    Frazier, A. Lindsay
    Messinger, Yoav
    Hill, D. Ashley
    GYNECOLOGIC ONCOLOGY, 2017, 147 (03) : 521 - 527