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Eculizumab, poor response to

MedGen UID:
816732
•Concept ID:
C3810402
•
Disease or Syndrome
Synonym: ECULIZUMAB, POOR RESPONSE TO
 
Gene (location): C5 (9q33.2)
 
OMIM®: 615749

Professional guidelines

PubMed

Thrombotic microangiopathy caused by methionine synthase deficiency: diagnosis and treatment pitfalls.
Vaisbich MH, Braga A, Gabrielle M, Bueno C, Piazzon F, Kok F
Pediatr Nephrol 2017 Jun;32(6):1089-1092. Epub 2017 Feb 16 doi: 10.1007/s00467-017-3615-6. PMID: 28210839

Recent clinical studies

Etiology

Antiviral cellular therapy for enhancing T-cell reconstitution before or after hematopoietic stem cell transplantation (ACES): a two-arm, open label phase II interventional trial of pediatric patients with risk factor assessment.
Keller MD, Hanley PJ, Chi YY, Aguayo-Hiraldo P, Dvorak CC, Verneris MR, Kohn DB, Pai SY, Dávila Saldaña BJ, Hanisch B, Quigg TC, Adams RH, Dahlberg A, Chandrakasan S, Hasan H, Malvar J, Jensen-Wachspress MA, Lazarski CA, Sani G, Idso JM, Lang H, Chansky P, McCann CD, Tanna J, Abraham AA, Webb JL, Shibli A, Keating AK, Satwani P, Muranski P, Hall E, Eckrich MJ, Shereck E, Miller H, Mamcarz E, Agarwal R, De Oliveira SN, Vander Lugt MT, Ebens CL, Aquino VM, Bednarski JJ, Chu J, Parikh S, Whangbo J, Lionakis M, Zambidis ET, Gourdine E, Bollard CM, Pulsipher MA
Nat Commun 2024 Apr 18;15(1):3258. doi: 10.1038/s41467-024-47057-2. PMID: 38637498Free PMC Article
Use of Eculizumab in Pediatric Patients with High-Risk Transplantation-Associated Thrombotic Microangiopathy: Outcomes and Risk Factors Associated with Response and Survival. A Retrospective Study on Behalf of the Spanish Group for Hematopoietic Transplantation and Cellular Therapy (GETH-TC).
Benítez Carabante MI, Bueno D, Alonso García L, López Torija I, Marsal J, Fernandez Navarro JM, Uria Oficialdegui ML, Panesso M, Molina B, Beléndez Bieler C, Palomo P, Pérez Martínez A, Diaz-de-Heredia C
Transplant Cell Ther 2024 Jun;30(6):601.e1-601.e13. Epub 2024 Mar 21 doi: 10.1016/j.jtct.2024.03.019. PMID: 38521410
Use of eculizumab in children with allogeneic haematopoietic stem cell transplantation associated thrombotic microangiopathy - a multicentre retrospective PDWP and IEWP EBMT study.
Svec P, Elfeky R, Galimard JE, Higham CS, Dalissier A, Quigg TC, Bueno Sanchez D, Han Lum S, Faraci M, Cole T, Pichler H, Benítez-Carabante MI, Horakova J, Gonzalez-Vicent M, Yanir A, Fagioli F, Wölfl M, von der Weid N, Protheroe R, Krivan G, Speckmann C, James B, Avcin SL, Bertrand Y, Verna M, Riha P, Patrick K, Cesaro S, Kalwak K, Bierings M, Büchner J, Mellgren K, Prohászka Z, Neven B, Lankester A, Corbacioglu S
Bone Marrow Transplant 2023 Feb;58(2):129-141. Epub 2022 Nov 4 doi: 10.1038/s41409-022-01852-x. PMID: 36333550
Transplantation-Associated Thrombotic Microangiopathy Risk Stratification: Is There a Window of Opportunity to Improve Outcomes?
Jodele S, Dandoy CE, Sabulski A, Koo J, Lane A, Myers KC, Wallace G, Chima RS, Teusink-Cross A, Hirsch R, Ryan TD, Benoit S, Davies SM
Transplant Cell Ther 2022 Jul;28(7):392.e1-392.e9. Epub 2022 Apr 29 doi: 10.1016/j.jtct.2022.04.019. PMID: 35490975Free PMC Article
Is complement blockade an acceptable therapeutic strategy for hematopoietic cell transplant-associated thrombotic microangiopathy?
Dhakal P, Bhatt VR
Bone Marrow Transplant 2017 Mar;52(3):352-356. Epub 2016 Oct 24 doi: 10.1038/bmt.2016.253. PMID: 27775697

Diagnosis

Use of Eculizumab in Pediatric Patients with High-Risk Transplantation-Associated Thrombotic Microangiopathy: Outcomes and Risk Factors Associated with Response and Survival. A Retrospective Study on Behalf of the Spanish Group for Hematopoietic Transplantation and Cellular Therapy (GETH-TC).
Benítez Carabante MI, Bueno D, Alonso García L, López Torija I, Marsal J, Fernandez Navarro JM, Uria Oficialdegui ML, Panesso M, Molina B, Beléndez Bieler C, Palomo P, Pérez Martínez A, Diaz-de-Heredia C
Transplant Cell Ther 2024 Jun;30(6):601.e1-601.e13. Epub 2024 Mar 21 doi: 10.1016/j.jtct.2024.03.019. PMID: 38521410
Utilizing therapeutic drug monitoring to optimize therapy with eculizumab and mycophenolate mofetil in a child with C3 glomerulonephritis.
Riedl Khursigara M, Chung E, Tjon J, Noone D, Chami R, Licht C, Teoh CW
Pediatr Nephrol 2023 Oct;38(10):3483-3487. Epub 2023 Mar 8 doi: 10.1007/s00467-023-05927-9. PMID: 36884075
Use of eculizumab in children with allogeneic haematopoietic stem cell transplantation associated thrombotic microangiopathy - a multicentre retrospective PDWP and IEWP EBMT study.
Svec P, Elfeky R, Galimard JE, Higham CS, Dalissier A, Quigg TC, Bueno Sanchez D, Han Lum S, Faraci M, Cole T, Pichler H, Benítez-Carabante MI, Horakova J, Gonzalez-Vicent M, Yanir A, Fagioli F, Wölfl M, von der Weid N, Protheroe R, Krivan G, Speckmann C, James B, Avcin SL, Bertrand Y, Verna M, Riha P, Patrick K, Cesaro S, Kalwak K, Bierings M, Büchner J, Mellgren K, Prohászka Z, Neven B, Lankester A, Corbacioglu S
Bone Marrow Transplant 2023 Feb;58(2):129-141. Epub 2022 Nov 4 doi: 10.1038/s41409-022-01852-x. PMID: 36333550
Thrombotic Microangiopathy Secondary to Systemic Sclerosis with Severe Complement Activation Not Responsive to Eculizumab: A Case Report.
Nakamura M, Abe T, Ochiai H
Am J Case Rep 2022 Jun 7;23:e936116. doi: 10.12659/AJCR.936116. PMID: 35671252Free PMC Article
Is complement blockade an acceptable therapeutic strategy for hematopoietic cell transplant-associated thrombotic microangiopathy?
Dhakal P, Bhatt VR
Bone Marrow Transplant 2017 Mar;52(3):352-356. Epub 2016 Oct 24 doi: 10.1038/bmt.2016.253. PMID: 27775697

Therapy

Use of Eculizumab in Pediatric Patients with High-Risk Transplantation-Associated Thrombotic Microangiopathy: Outcomes and Risk Factors Associated with Response and Survival. A Retrospective Study on Behalf of the Spanish Group for Hematopoietic Transplantation and Cellular Therapy (GETH-TC).
Benítez Carabante MI, Bueno D, Alonso García L, López Torija I, Marsal J, Fernandez Navarro JM, Uria Oficialdegui ML, Panesso M, Molina B, Beléndez Bieler C, Palomo P, Pérez Martínez A, Diaz-de-Heredia C
Transplant Cell Ther 2024 Jun;30(6):601.e1-601.e13. Epub 2024 Mar 21 doi: 10.1016/j.jtct.2024.03.019. PMID: 38521410
Utilizing therapeutic drug monitoring to optimize therapy with eculizumab and mycophenolate mofetil in a child with C3 glomerulonephritis.
Riedl Khursigara M, Chung E, Tjon J, Noone D, Chami R, Licht C, Teoh CW
Pediatr Nephrol 2023 Oct;38(10):3483-3487. Epub 2023 Mar 8 doi: 10.1007/s00467-023-05927-9. PMID: 36884075
Use of eculizumab in children with allogeneic haematopoietic stem cell transplantation associated thrombotic microangiopathy - a multicentre retrospective PDWP and IEWP EBMT study.
Svec P, Elfeky R, Galimard JE, Higham CS, Dalissier A, Quigg TC, Bueno Sanchez D, Han Lum S, Faraci M, Cole T, Pichler H, Benítez-Carabante MI, Horakova J, Gonzalez-Vicent M, Yanir A, Fagioli F, Wölfl M, von der Weid N, Protheroe R, Krivan G, Speckmann C, James B, Avcin SL, Bertrand Y, Verna M, Riha P, Patrick K, Cesaro S, Kalwak K, Bierings M, Büchner J, Mellgren K, Prohászka Z, Neven B, Lankester A, Corbacioglu S
Bone Marrow Transplant 2023 Feb;58(2):129-141. Epub 2022 Nov 4 doi: 10.1038/s41409-022-01852-x. PMID: 36333550
Is complement blockade an acceptable therapeutic strategy for hematopoietic cell transplant-associated thrombotic microangiopathy?
Dhakal P, Bhatt VR
Bone Marrow Transplant 2017 Mar;52(3):352-356. Epub 2016 Oct 24 doi: 10.1038/bmt.2016.253. PMID: 27775697
Genetic variants in C5 and poor response to eculizumab.
Nishimura J, Yamamoto M, Hayashi S, Ohyashiki K, Ando K, Brodsky AL, Noji H, Kitamura K, Eto T, Takahashi T, Masuko M, Matsumoto T, Wano Y, Shichishima T, Shibayama H, Hase M, Li L, Johnson K, Lazarowski A, Tamburini P, Inazawa J, Kinoshita T, Kanakura Y
N Engl J Med 2014 Feb 13;370(7):632-9. doi: 10.1056/NEJMoa1311084. PMID: 24521109

Prognosis

D-dimer and sinusoidal obstructive syndrome-novel poor prognostic features of thrombotic microangiopathy in children after hematopoietic cellular therapy in a single institution prospective cohort study.
Schoettler ML, French K, Harris A, Bryson E, Deeb L, Hudson Z, Obordo J, Chandrakasan S, Parikh S, Watkins B, Stenger E, Qayed M, Chonat S, Westbrook A, Switchenko J, Williams KM
Am J Hematol 2024 Mar;99(3):370-379. Epub 2024 Jan 2 doi: 10.1002/ajh.27186. PMID: 38164997
Thrombotic Microangiopathy Secondary to Systemic Sclerosis with Severe Complement Activation Not Responsive to Eculizumab: A Case Report.
Nakamura M, Abe T, Ochiai H
Am J Case Rep 2022 Jun 7;23:e936116. doi: 10.12659/AJCR.936116. PMID: 35671252Free PMC Article
Two cases of carfilzomib-induced thrombotic microangiopathy successfully treated with Eculizumab in multiple myeloma.
Rassner M, Baur R, Wäsch R, Schiffer M, Schneider J, Mackensen A, Engelhardt M
BMC Nephrol 2021 Jan 18;22(1):32. doi: 10.1186/s12882-020-02226-5. PMID: 33461512Free PMC Article
Severe, persistent neurotoxicity after transplant-associated thrombotic microangiopathy in a pediatric patient despite treatment with eculizumab.
Schoettler M, Duncan C, Lehmann L
Pediatr Transplant 2019 May;23(3):e13381. Epub 2019 Mar 3 doi: 10.1111/petr.13381. PMID: 30828947Free PMC Article
Genetic variants in C5 and poor response to eculizumab.
Nishimura J, Yamamoto M, Hayashi S, Ohyashiki K, Ando K, Brodsky AL, Noji H, Kitamura K, Eto T, Takahashi T, Masuko M, Matsumoto T, Wano Y, Shichishima T, Shibayama H, Hase M, Li L, Johnson K, Lazarowski A, Tamburini P, Inazawa J, Kinoshita T, Kanakura Y
N Engl J Med 2014 Feb 13;370(7):632-9. doi: 10.1056/NEJMoa1311084. PMID: 24521109

Clinical prediction guides

D-dimer and sinusoidal obstructive syndrome-novel poor prognostic features of thrombotic microangiopathy in children after hematopoietic cellular therapy in a single institution prospective cohort study.
Schoettler ML, French K, Harris A, Bryson E, Deeb L, Hudson Z, Obordo J, Chandrakasan S, Parikh S, Watkins B, Stenger E, Qayed M, Chonat S, Westbrook A, Switchenko J, Williams KM
Am J Hematol 2024 Mar;99(3):370-379. Epub 2024 Jan 2 doi: 10.1002/ajh.27186. PMID: 38164997
Use of eculizumab in children with allogeneic haematopoietic stem cell transplantation associated thrombotic microangiopathy - a multicentre retrospective PDWP and IEWP EBMT study.
Svec P, Elfeky R, Galimard JE, Higham CS, Dalissier A, Quigg TC, Bueno Sanchez D, Han Lum S, Faraci M, Cole T, Pichler H, Benítez-Carabante MI, Horakova J, Gonzalez-Vicent M, Yanir A, Fagioli F, Wölfl M, von der Weid N, Protheroe R, Krivan G, Speckmann C, James B, Avcin SL, Bertrand Y, Verna M, Riha P, Patrick K, Cesaro S, Kalwak K, Bierings M, Büchner J, Mellgren K, Prohászka Z, Neven B, Lankester A, Corbacioglu S
Bone Marrow Transplant 2023 Feb;58(2):129-141. Epub 2022 Nov 4 doi: 10.1038/s41409-022-01852-x. PMID: 36333550
Transplantation-Associated Thrombotic Microangiopathy Risk Stratification: Is There a Window of Opportunity to Improve Outcomes?
Jodele S, Dandoy CE, Sabulski A, Koo J, Lane A, Myers KC, Wallace G, Chima RS, Teusink-Cross A, Hirsch R, Ryan TD, Benoit S, Davies SM
Transplant Cell Ther 2022 Jul;28(7):392.e1-392.e9. Epub 2022 Apr 29 doi: 10.1016/j.jtct.2022.04.019. PMID: 35490975Free PMC Article
Genetic variants in C5 and poor response to eculizumab.
Nishimura J, Yamamoto M, Hayashi S, Ohyashiki K, Ando K, Brodsky AL, Noji H, Kitamura K, Eto T, Takahashi T, Masuko M, Matsumoto T, Wano Y, Shichishima T, Shibayama H, Hase M, Li L, Johnson K, Lazarowski A, Tamburini P, Inazawa J, Kinoshita T, Kanakura Y
N Engl J Med 2014 Feb 13;370(7):632-9. doi: 10.1056/NEJMoa1311084. PMID: 24521109

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