ABSTRACT
Justification: In India, there is a lack of uniformity of treatment strategies for aplastic anemia (AA), and many children are managed only with supportive care due to non-availability of hematopoietic stem cell transplantation (HSCT). Process: Eminent national faculty members were invited to participate in the process of forming a consensus statement in Hyderabad in July, 2016. Draft guidelines were circulated to all members, and comments received in a online meeting in October, 2020 were incorporated into the final draft. These were approved by all experts. Objective: To facilitate appropriate management of children with acquired aplastic anemia. Recommendations: Key recommendations are: i) A bone marrow biopsy is must to make a diagnosis of AA; ii) Rule out inherited bone marrow failure syndromes (IBMFS), connective tissue disorders, viral infections, paroxysmal nocturnal hemoglobinuria (PNH), drug or heavy metal induced marrow suppression in all cases of AA; iii) Conservative approach to transfusions should be followed, with a target to keep hemoglobin >6 g/dL in children with no co-morbidities; iv) HLA-matched sibling donor HSCT is the preferred choice of treatment for newly diagnosed very severe/ severe AA; v) In absence of HLA-matched family donor, a matched unrelated donor (MUD) transplant or immunosuppressive therapy (IST) should be considered as alternate choice based on physician expertise; vi) Fludarabine, cyclophosphamide and anti-thymocyte globulin (ATG) based conditioning with cyclosporine and methotrexate as graft versus host disease (GvHD) prophylaxis is the preferred regimen; vii) Horse ATG and cyclosporine are the recommended drugs for IST. One should wait for 3-6 months for the response assessment and consideration of next line therapy.
ABSTRACT
Justification: Practitioners and people need information about the therapeutic potential of umbilical cord blood stem cells and pros andcons of storing cord blood in public versus private banks.Process: Indian Academy of Pediatrics conducted a consultative meeting on umbilical cord blood banking on 25th June 2016 in Pune,attended by experts in the field of hematopoietic stem cell transplantation working across India. Review of scientific literature was alsoperformed. All expert committee members reviewed the final manuscript.Objective: To bring out consensus guidelines for umbilical cord banking in India.Recommendations: Umbilical cord blood stem cell transplantation has been used to cure many malignant disorders, hematologicalconditions, immune deficiency disorders and inherited metabolic disorders, even when it’s partially HLA mismatched. Collectionprocedure is safe for mother and baby in an otherwise uncomplicated delivery. Public cord blood banking should be promoted over privatebanking. Private cord blood banking is highly recommended when an existing family member (sibling or biological parent) is sufferingfrom diseases approved to be cured by allogenic stem cell transplantation. Otherwise, private cord blood banking is not a ‘biologicalinsurance’, and should be discouraged. At present, autologous cord stem cells cannot be used for treating diseases of genetic origin,metabolic disorders and hematological cancers. Advertisements for private banking are often misleading. Legislative measures arerequired to regularize the marketing strategies of cord blood banking.