Hematopoietic Stem Cell Transplantation in Paroxysmal Nocturnal Hemoglobinuria

Abstract

BACKGROUND: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired hematological disorder characterized by episodic intravascular hemolysis. Despite newer therapies such as eculizumab, hematopoietic stem cell transplantation (HSCT) remains the only curative therapy. MATERIALS AND METHODS: An observational analytical retrospective study was conducted comprising eight PNH patients who had undergone HSCT at our center. From January 2014 to December 2020, we performed HSCT in 8 PNH patients, 5 female and 3 male, with median age of 32 years (range: 15–38 years). RESULTS: Seven patients had pancytopenia, associated with marrow hypocellularity. One patient presented with a thrombotic episode. Seven had matched sibling donor (MSD) transplant and one underwent haploidentical transplant. The median time from diagnosis to transplant was 7 months (range: 1–38 months). All received reduced intensity conditioning (RIC): Flu-Bu-Cy-A TG for MSD transplants and Flu-A TG-Post Cy in haploidentical transplants. Median CD34+ stem cell dose was 5.5 × 106/kg (range 3.4–5.8 × 106/kg). All had successful engraftment. Acute graft versus host disease (GVHD) Grade I–III occurred in six patients. Chronic GVHD occurred in two patients. One patient succumbed to Grade III liver GVHD. Rest seven patients are alive till date and transfusion independent. The overall survival was 87.5% at a median of 26 months. CONCLUSIONS: HSCT is the only curative therapy for PNH. With the use of RIC, the outcomes of PNH patients can be improved. The incidence of GVHD is high with RIC regimens but can be managed with prompt immunosuppression.

Procalcitonin elevation in febrile recipients during pre-transplant conditioning with anti-thymocyte globulin

“…Among the 76 patients who developed fever during the conditioning stage and had their PCT levels measured, 48 were PCT-negative and 28 were PCT-positive ( Table 1 ). Although it is recognized that ATG administration in conditioning regimens for allo-HSCT leads to elevated PCT levels 13 , 14 , there is a lack of reported data on PCT levels in “ febrile patients ” following ATG administration. Therefore, the impact of ATG use on PCT positivity during pre-transplantation procedures in the context of fever was investigated.…”

“…Therefore, it is important to distinguish these symptoms from those of infectious diseases. Although ATG administration in allo-HSCT conditioning regimens is known to increase PCT levels 13 , 14 , the PCT levels in febrile patients following ATG administration have not yet been reported. Furthermore, no study has directly compared the increase in PCT levels during conditioning for allo-HSCT and its risk factors between ATG and non-ATG regimens.…”