Related Experiment Video
Updated: May 4, 2026
Intrasplenic Transplantation of Hepatocytes After Partial Hepatectomy in NOD.SCID Mice
Published on: February 10, 2018
Hemophagocytic lymphohistiocytosis in infants: a single center experience from India
Mohammed Ramzan1, Satya Prakash Yadav, Gaurav Kharya
11Pediatric Hematology Oncology & Bone Marrow Transplant Unit, Department of Pediatrics, Institute of Child Health, Sir Ganga Ram Hospital , Old Rajinder Nagar, New Delhi , India.
Insights
Outcome data for infant hemophagocytic lymphohistiocytosis (HLH) in India is scarce. Stem cell transplant (SCT) is feasible for familial HLH in developing nations, but challenges like sepsis persist.
Area of Science:
- Pediatric Hematology
- Immunology
- Genetics
Background:
- Limited outcome data exists for infant hemophagocytic lymphohistiocytosis (HLH) in India, particularly after stem cell transplantation (SCT).
- This study presents outcome data for eight infants diagnosed with HLH in India.
Observation:
- The mean age of infants was 7.1 months.
- Mutation analysis revealed genetic causes in seven patients, including Griscelli syndrome and homozygous mutations in Perforin, Munc, and STX11 genes.
- Three patients had no identifiable mutations.
Findings:
- All patients were treated according to the HLH 2004 protocol.
- Four infants died during induction therapy, and one abandoned treatment.
- Two infants underwent SCT; one is awaiting SCT.
- One patient achieved disease-free survival 22 months post-SCT with a matched sibling donor.
- Another patient died 5 months post-unrelated double cord blood transplant due to renal failure.
Implications:
- Stem cell transplantation (SCT) is a feasible treatment option for infants with familial HLH in developing countries.
- Significant barriers, including sepsis and disease refractoriness, must be addressed to improve SCT outcomes in this population.
Abstract:
There is paucity of outcome data for hemophagocytic lymphohistiocytosis (HLH) in infants from India, especially post stem cell transplant (SCT). We report outcome data of eight infants diagnosed with HLH. Mean age was 7.1 months (range 2-11). Mutation analysis was possible in seven patients. One patient had Griscelli syndrome. In three patients, no known mutation could be identified, while in remaining three homozygous mutations in Perforin, Munc and STX11 gene were identified. All were treated as per HLH 2004 protocol. Four died during induction phase. One patient abandoned therapy. Two underwent SCT, while one is awaiting SCT. First patient is alive and disease-free at 22 months postmatched sibling donor SCT. Second underwent unrelated double cord blood transplant, but died 5 months posttransplant due to renal failure. It is feasible to offer SCT for infants with familial HLH in the developing world although barriers like sepsis and disease refractoriness remain.
