Successful bone marrow transplantation for severe aplastic anemia following orthotopic liver transplantation: long-term follow-up and outcome

Abstract

Summary:Severe aplastic anemia (SAA) is well described in children following liver transplantation for fulminant hepatic failure (FHF) secondary to non-A, non-B, non-C hepatitis, and is associated with a high mortality rate. Successful immunosuppressive treatment of SAA following liver transplantation has been reported, but death from infectious complications is not uncommon. We report the 8-year follow-up of a 3.5-year-old boy who underwent successful HLA-identical sibling donor bone marrow transplant for SAA 7 months following orthotopic liver transplant for non-A, non-B, non-C hepatitis. His post-bone marrow transplantation course was uneventful with no evidence of liver toxicity. Eight months following BMT he developed renal cell carcinoma metastatic to lymph nodes which was treated surgically. Six years following BMT he developed a mucoepidermoid carcinoma of the parotid gland also treated surgically. Despite these malignancies, he is currently well 8 years following liver and bone marrow transplantation, without signs of GVHD, growth failure or liver graft rejection. This is the first report of long-term follow-up of bone marrow transplantation for SAA following liver transplantation. The occurrence of two subsequent malignancies in this child underscores the need for close follow-up of future similar cases. Bone Marrow Transplantation (2001) 28, 523-526.

Hematopoietic stem-cell transplantation following solid-organ transplantation in children

“…6 In the case report with the longest follow-up, the patient had severe complications following HSCT with development of metastatic renal cell carcinoma and a mucoepidermoid carcinoma of the parotid gland both of which were treated surgically. 5 The stem-cell donors for our patients were different from these previous reports with one patient receiving a 10 out of 10 HLA matched living-unrelated donor and the other a haploidentical donor. Both of the patients engrafted, but succumbed to multi-organ failure and infections at 1 and 6 months post-HSCT.…”

“…29 However, successful allogeneic HSCT in pediatric patients developing SAA following orthotopic liver transplantation has been previously described only in case reports. [2][3][4][5][6]30 All the reported HSCT utilized matched living-related donors. The case reports had very short follow-up and no reported complications.…”

“…[1][2][3][4][5][6] However, those publications likely reflect a positive reporting bias, as no reports of a cohort have been published to date. The need for complete cohort studies or prospective studies of such transplants is essential for clinicians to be able to understand appropriate indications, complications and outcomes.…”

“…Indications for HSCT after solid-organ transplantation that have been reported include mainly severe aplastic anemia (SAA) following liver transplantation. [2][3][4][5][6] Post transplant lymphoproliferative disease (PTLD) is an important cause of morbidity and mortality after solid-organ or stem-cell transplantation, with a reported incidence of 1-10% in all adult transplant recipients; this number has been reported to be even higher in children. [7][8][9][10][11][12][13] PTLD represents a spectrum of lymphoid proliferation that can progress to monoclonal malignant lymphoma.…”

Should we be performing more combined hematopoietic stem cell plus solid organ transplants?

“…All seven patients received myeloablative conditioning regimens prior to BMT and were alive at 4 months to 8 years post-BMT. The report of the patient with the longest follow-up 54 demonstrates the importance of close monitoring in the post-transplant immunocompromised phase. This patient developed renal cell carcinoma and mucoepidermoid carcinoma of the parotid gland at 8 months and 6 years post-BMT, respectively.…”