Treatment options for PTLD

Treatment options for PTLD include the following:

First-line therapy for patients with PTLD is to reduce immunosuppressive therapy as much as possible.[27,28] However, this may not be possible because of the increased risk of organ rejection or graft-versus-host disease (GVHD).

Rituximab, an anti-CD20 antibody, has been used in the posttransplant setting. Rituximab as a single agent to treat PTLD after organ transplant has demonstrated efficacy in adult patients, but data are lacking in pediatric patients.

Evidence (rituximab):

For more information, see the Polymorphic Posttransplant Lymphoproliferative Disorder section in Aggressive B-Cell Non-Hodgkin Lymphoma Treatment.

Low-intensity chemotherapy has been effective in patients with EBV-positive, CD20-positive B-lineage PTLD.[19,29] An event-free survival (EFS) rate of 67% was demonstrated in a Children's Oncology Group study using rituximab plus cyclophosphamide and prednisone in children with PTLD after solid organ transplant in whom immune suppression was reduced.[19][Level of evidence B4]

Some studies suggest that modified conventional lymphoma therapy is effective for patients who have PTLD with MYC translocations and Burkitt lymphoma histology.[24,25][Level of evidence C2] A multicenter retrospective review summarized the treatments and outcomes of 35 patients with PTLD-BL. Fluorescence in situ hybridization (FISH) detected the MYC translocation in 95% of cases. Treatments ranged from rituximab only to FAB/LMB therapy. The 3-year EFS and OS rates for all patients were 66.2% and 88.0%, respectively. The most commonly used therapy was a low-dose chemotherapy approach that is similar to the COG regimen (cyclophosphamide, prednisone, and rituximab [CPR]; n = 13). Using this approach, the EFS rate was 52.7%, and the OS rate was 84.6%.[26]

Patients with T-cell or Hodgkin-like PTLD are usually treated with standard lymphoma-specific chemotherapy regimens.[30-33]

Antirejection therapy is usually decreased or discontinued when chemotherapy is given to avoid excessive toxicity. There are no data to guide the re-initiation of immunosuppressive therapy after chemotherapy treatment. There is little evidence of benefit for chemotherapy after HSCT.

Adoptive immunotherapy with either donor lymphocytes or ex vivo–generated EBV-specific cytotoxic T lymphocytes (EBV-CTLs) has been effective in treating patients with PTLD after blood or bone marrow transplant.[34,35] To make this approach more broadly applicable, banks of off-the-shelf, third-party, allogeneic EBV-CTLs derived from healthy donors have been developed.[36,37] EBV-CTLs were evaluated in 46 patients with PTLD that had either progressed during rituximab treatment, not fully responded to rituximab treatment, or had recurred after a previous response. The following results were observed:[38]