Dr Anupama Rao  

Consultant in Paediatric Haematology 
Dr Anupama Rao  
Dr Anupama Rao  


Dr Anupama Rao is a Paediatric Haematologist at Great Ormond Street Hospital for Children (GOSH). She has extensive experience and a breadth of knowledge in the management of children with rare haematological diseases such as Juvenile Myelomonocytic Leukaemia (JMML), Myelodysplastic Syndromes (MDS), Familial Myelodysplasia, GATA2, MonoMac, Emberger Syndromes and Bone Marrow Failure Syndromes.  

Dr Rao leads a national UK wide study as Chief Investigator on Paediatric Myelodysplasia and Marrow Failure with the goal of enhanced access to advanced diagnostic next generation sequencing testing, genotype-clinical phenotype correlation and develop personalized treatment strategies for children with rare marrow failure syndromes. She has experience in treating children on early phase clinical trials with a proven benefit which has led to improved outcomes and successful bone marrow transplantation in children with rare diseases.   

Dr Rao also has specialist clinical experience in haemophagocytic lymphohistiocytosis (HLH), specifically the diagnosis of genetic HLH. She is the UK Principal Investigator for a novel treatment in HLH specifically in the management of refractory HLH.   

Dr Rao is committed to improving long term outcomes of children with rare diseases to ensure they lead long and healthy lives. With clinical experience in the long term effects of bone marrow transplantation on growth and puberty Dr Rao ensures that she provide holistic care for siblings and the family of a child with a rare haematological disease.  

Languages Spoken:

  • English
  • Hindi


Dr Rao has specialist expertise is in the following areas:  

  • Diagnosis and management of children with rare haematological diseases ie. JMML, Myelodysplastic Syndromes, Bone Marrow Failure Syndromes, Familial Myelodysplasia 
  • Access to and interpretation of comprehensive next generation sequencing panels relevant to JMML/marrow failure disorders 
  • Long Term follow Up of children following bone marrow transplantation for JMML/ Marrow Failure Disorders 
  • Diagnosis of genetic primary HLH and management of children relapsing or refractory to HLH 
  • Growth and pubertal surveillance and management following bone marrow transplantation 
  • Paediatric haematology service (both inpatient and outpatient care) for acute lymphoblastic leukaemia and acute myeloid leukaemia
  • Lead on the haematology endocrinology service, reviewing growth and pubertal surveillance following bone marrow transplantation 
  • Neurocognitive outcomes following CNS directed therapy in leukaemia and following bone marrow transplantation in HLH  
  • Member of the Royal College of Paediatrics and Child Health 
  • Member of the Royal College of Pathologists 
  • Member of the British Society of Haematology  
  • Member of the Childhood Cancer and Leukaemia Group 
  • Genomic architecture of pre-malignant myeloid diseases ie. Myelodysplastic Syndromes and Inherited Bone Marrow Failure Syndromes  
  • Prognostic risk stratification based on clinical and molecular genomics 
  • Stem cell biology in JMML  
  • Cellular architecture of Familial Myelodysplastic Syndromes  
  • Translational research to identify novel therapeutic targets for better disease outcomes in children with rare diseases

News & Publications

Cruikshank M, Anoop P, Nikolajeva O, Rao A, Rao K, Gilmour K, Eleftheriou D, Brogan PA. 2015. Screening assays for primary haemophagocytic lymphohistiocytosis in children presenting with suspected macrophage activation syndrome. Pediatr Rheumatol Online J.   

Jackson J, Titman P, Butler S, Bond K, Rao A, Veys P, Chiesa R, Leiper A, Riley L, Gilmour K, Amrolia P, Rao K. 2013. Cognitive and psychosocial function post hematopoietic stem cell transplantation in children with hemophagocytic lymphohistiocytosis. J Allergy Clin Immunol. 132(4) pp. 889-95.  

Zeilhofer U, Ashworth M, Amrolia P, Rao A, Chiesa R, Veys P, Rao K. 2013. Pulmonary hypertension following haematopoietic stem cell transplantation for primary haemophagocytic lymphohistiocytosis. Pediatr Blood Cancer. 60(3) pp. 521-3.   

Hiwarkar P, Rao A. 2011. Revisiting Darwinism explains extinction of fetal erythroid progenitors in a leukaemogenic model of a paediatric myeloproliferative neoplasm. Br J Haematol. 155(1). 

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