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Haploidentical Hematopoietic Stem Cell Transplant Complicated by Atypical Hemolytic Uremic Syndrome and Kidney Transplant From the Same Donor With No Immunosuppression but C5 Inhibition

Ardissino, Gianluigi, MD, PhD1; Cresseri, Donata, MD2; Giglio, Fabio, MD3; Onida, Francesco, MD4; Iannuzzella, Francesco, MD5; Tel, Francesca, MD1; Giussani, Antenore, MD6; Messa, Piergiorgio, MD7; Longhi, Selena, MD1; Vincenti, Daniele, MD8; Tedeschi, Silvana, MD9; Cugno, Massimo, MD10; Ciceri, Fabio, MD11

doi: 10.1097/TP.0000000000002505
Original Clinical Science—General

Background Atypical hemolytic uremic syndrome (aHUS) is life-threatening condition particularly when complicating allograft hematopoietic stem cell transplant (HSCT). In the past, the outcome was very poor with the majority of patients reaching end-stage renal disease or dying with little or no chances of kidney transplant (KTx) due to the high risk of relapse. The availability of C5 inhibition has opened up significant therapeutic opportunities and has improved the outcome particularly if complement dysregulation (CD) is the underlying pathogenetic mechanism.

Methods We describe a peculiar case of a girl with aHUS complicating HSCT and her subsequent successful KTx received from the same donor thus performed without immunosuppression but anti-C5 inhibition.

Results Soon after HSCT performed for acute lymphoblastic leukemia, the patient developed a TMA due to CD and reached end-stage renal disease. After 2 years on dialysis, the patient received a KTx from her father who was already the HSCT donor. Given the full chimerism, no immunosuppressive agent was prescribed except a short (2 days) course of steroids and eculizumab to prevent aHUS relapse. Nine months after the KTx, the patient is well with normal renal function, no immunosuppression and continues eculizumab prevention of aHUS (1 infusion every 21 days).

Conclusions All patients with transplant-associated thrombotic microangiopathy should be screened for the causes of CD. C5 inhibition with eculizumab is an important therapeutic resource to manage this complication. When KTx is necessary, immunosuppression can be safely withhold in case of same donor for both grafts and documented full chimerism.

This complex case describes a patient with B cell acute lymphoblastic leukemia treated with 2 hematopoietic stem cell transplants: the first, a matched unrelated, the second, a haplo-identical transplant from her father complicated by atypical-hemolytic uremic syndrome (aHUS) leading to end-stage renal disease followed by a kidney transplant without allo-immunosuppression, but reduced eculizumab to prevent aHUS caused by a putative autoantibody.

1 Center for HUS Prevention, Control and Management at the Pediatric Nephrology, Dialysis and Transplantation Unit, Fondazione IRCCS Ca’ Granda, Ospedale Maggiore Policlinico, Milan, Italy.

2 Center for HUS Prevention, Control and Management at the Nephrology, Dialysis and Kidney Transplantation Unit, Fondazione IRCCS Ca’ Granda, Ospedale Maggiore Policlinico, Milan, Italy.

3 Hematology and Bone Marrow Transplantation Unit, IRCCS S. Raffaele Scientific Institution, Milan. Italy.

4 Oncology and Hemato-Oncology Department, University of Milan, BTM Center, Fondazione IRCCS Ca’ Granda, Ospedale Maggiore Policlinico, Milan, Italy.

5 Nephrology and Dialysis Unit, IRCSS Arcispedale S. Maria Nuova, Reggio Emilia, Italy.

6 Center for HUS Prevention, Control and Management at the Kidney Transplant Unit, Fondazione IRCCS Ca’ Granda, Ospedale Maggiore Policlinico, Milan, Italy.

7 Dipartimento di Scienze Cliniche e di Comunità, Università degli Studi di Milano, Milan, Italy.

8 UOC Coordinamento Trapianto (NITp), Dip. Servizi e Medicina Preventiva, Fondazione IRCCS Ca’ Granda, Ospedale Maggiore Policlinico, Milan, Italy.

9 Center for HUS Prevention, Control and Management at the Laboratory of Medical Genetics, Fondazione IRCCS Ca’ Granda, Ospedale Maggiore Policlinico, Milan, Italy.

10 Center for HUS Prevention, Control and Management at the Internal Medicine, University of Milan, Fondazione IRCCS Ca’ Granda, Ospedale Maggiore Policlinico, Milan, Italy.

11 Hematology and Bone Marrow Transplantation Unit, IRCCS S. Raffaele Scientific Institution, Milan, Italy.

Received 5 March 2018. Revision received 26 September 2018.

Accepted 2 October 2018.

The authors declare no funding or conflicts of interest.

G.A. participated in the writing of the article. D.C. participated in the writing of the article. F.G. participated in the writing of the article. F.O. participated in the writing of the article. F.I. participated in the writing of the article. F.T. participated in the writing of the article. A.G. participated in the writing of the article. P.M. participated in the writing of the article. S.L. participated in the writing of the article. D.V. participated in the writing of the article. S.T. participated in the writing of the article. M.C. participated in the writing of the article. F.C. participated in the writing of the article.

Correspondence: Gianluigi Ardissino, MD, PhD, Center for HUS Control, Prevention and Management, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Via Commenda 9, 20122 Milan, Italy. (ardissino@centroseu.org).

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