Safety and efficacy of Sofosbuvir and Velpatasvir in children with active hepatitis C virus infection undergoing haploidentical transplantation

Abstract

Direct Acting Antivirals (DAA) have changed the landscape of hepatitis C virus (HCV) infection with high cure rates across genotypes. However, the use of these agents in the setting of allogeneic hematopoietic cell transplantation (HCT) has been limited. In this context, we report the outcome of five children (5‐12 years) with relapsed and refractory leukemia and active HCV infection (genotype 1b), who underwent urgent haploidentical HCT and were treated with Sofosbuvir and Velpatasvir (Sof‐Vel) from initiation of treatment to 24 weeks post‐HCT. All achieved complete virologic response (VR) at a median of 2 weeks, with normalization of liver enzymes. There were no adverse events related to the use of Sof‐Vel, with no major fluctuations in cyclosporine levels. Two of the patients developed chronic GVHD and one relapsed. Sof‐Vel was continued in one of them along with sirolimus without affecting drug levels. With a median follow‐up of 15 months, four patients are disease free with sustained VR. Our study shows that combination of Sof‐Vel might be effective in inducing rapid complete and sustained VR during HCT without any major untoward drug interaction.

Efficacy and safety of treatment with sofosbuvir/velpatasvir in patients aged 6–18 years with chronic hepatitis C—Results of the PANDAA‐PED study

“…Jaiswal et al presented five cases of children (5–12 years) with leukaemia and active HCV infection (genotype 1b), who underwent urgent haematopoietic cell transplantation (HCT) and were treated with SOF/VEL. They all achieved SVR with normalization of liver enzymes 19 . This suggests that the treatment outcomes may be good even in immunocompromised children.…”

“…They all achieved SVR with normalization of liver enzymes. 19 This suggests that the treatment outcomes may be good even in immunocompromised children.…”

Defer no more: advances in the treatment and prevention of chronic hepatitis C virus infection in children

“…Early eradication of HCV is of paramount importance to prevent liver-related complications. Single patient case reports or case series of successful HCV treatment using DAA regimens have been reported in children with haematologic malignancies and those undergoing haematopoietic stem cell therapy [25,40,41 ▪ ,42,43 ▪ ,44]. Safety profiles were excellent, with no reported DAA-related serious adverse events, chemotherapy interruptions or significant toxicities due to drug interactions [25,40,41 ▪ ,42,43 ▪ ,44].…”

“…Early eradication of HCV is of paramount importance to prevent liver-related complications. Single patient case reports or case series of successful HCV treatment using DAA regimens have been reported in children with haematologic malignancies and those undergoing haematopoietic stem cell therapy [25,40,41…”

Breakthroughs and challenges in the management of pediatric viral hepatitis

“…They achieved stable viral clearance with calcineurin inhibitor-based regimens (cyclosporin + mycophenolate mofetil, and tacrolimus + sirolimus, respectively). The other five children were 5-12 years of age, and received haploidentical allogeneic HSCT for genotype 1b refractory lymphoblastic leukemia, and were under cyclosporin maintenance[ 55 ]. All of them were treated with 24 wk of sofosbuvir/velpatasvir after a median HSCT follow-up of 15 mo, and achieved SVR by 1 mo.…”