Hematopoietic stem cell transplantation
Orphan Drug Cold Chain RequiredStandard of care
Description
Allogeneic hematopoietic stem cell transplantation (HSCT) is the definitive curative treatment for T-B+ severe combined immunodeficiency (SCID), including forms caused by CD3 subunit deficiencies. HSCT replaces the defective immune system with functional donor stem cells that can differentiate into all blood cell lineages. Early transplantation before the onset of severe infections significantly improves outcomes in SCID patients.
Indications & Therapeutic Use
Severe combined immunodeficiency, primary immunodeficiency disorders, bone marrow failure syndromes
Global Availability (10 countries)
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Available Through
This drug is available for procurement through the following fulfilment partner.
Fulfilment Partner
Hematopoietic stem cell transplantation
| Generic Name | Hematopoietic stem cell transplantation |
| Active Ingredient | Allogeneic hematopoietic stem cells |
| Drug Class | Severe combined immunodeficiency |
| Manufacturer | Various transplant centers |
| Dosage Forms | Intravenous infusion of stem cells |
| Orphan Status | Yes — Orphan Drug |
| Cold Chain | Required |
| Lead Time | 90 days |
| Reg. Status | Standard of care |
| Clinical Trial | NCT00001467 |
| Countries | 10 countries |
Authority Signals
Last Verified3/21/2026
Data ConfidenceHigh (AI + Pharmacist)
Source Citations11 Validated Nodes