Diagnosis: Sickle cell anemia

Treatment: Outpatient visits for stem cell transplant evaluation and surgery

The insurer denied coverage for Outpatient visits for stem cell transplant evaluation and surgery

The denial is upheld

The patient is a male with a history of sickle cell anemia. The patient is currently on hydroxyurea and is doing well with no documentation of stroke, acute chest syndrome or multiple vaso-occlusive crises.

A letter of medical necessity, states that "He has been on hydroxyurea and though doing fairly well, he still suffers from 2-3 pain crisis each year."

The request is for outpatient visits for a potential allogeneic bone marrow transplant from a haploidentical or unrelated donor is the topic of this review.

The health care plan did act reasonably and with sound medical judgment and in the best interest of the patient. The use of matched sibling transplant has become the standard therapy for patients with sickle cell disease. Since the use of unrelated donors is associated with a higher risk of complications including chronic graft versus host disease its use is limited to patients with central nervous system (CNS) problems such as stroke and cerebral vasculopathy both to prevent the progression of the CNS disease as well as replace the need for chronic packed red blood cell transfusion or are developing antibodies.

Outpatient visits for stem cell transplant evaluation and surgery is not medically necessary for this patient. Hematopoietic stem cell transplantation (HSCT) is a curative treatment for sickle cell disease (SCD). Patients transplanted from a human leukocyte antigen (HLA) identical sibling have an overall survival (OS) that varies between 88% and up to 100% with the highest survival reported for younger patients and an event-free survival (EFS) estimated between 81 and 93%. Along with poor patient awareness, the lack of a suitable donor is a major barrier preventing the widespread application of HSCT in SCD patients. For patients lacking an HLA-identical sibling donor, the best treatment strategy is still controversial, and in addition to alternative donor transplant, other therapeutic approaches such as gene therapy or novel treatments to decrease disease symptoms and complications are emerging. At the present time the use of non-HLA matched sibling transplant is limited to patients with severe sickle cell anemia and would not be considered medically necessary for this patient with minimal complications of his sickle cell anemia.

"In conclusion, unrelated donor HSCT is a valid option for SCD patients who lack an HLA-identical sibling donor, preferably in the context of clinical trials. Using a 10/10 HLA-matched unrelated donor yields better survival indicating that HLA matching is an important donor selection factor in this nonmalignant disease." (Gluckman, E., et al., 2020).

"Unrelated donor (URD) hematopoietic cell transplantation (HCT) in children with sickle cell disease (SCD) is associated with a high incidence of rejection and graft-versus-host disease (GVHD)." (Rangarajan, H. G., 2018).

Finally, "For individuals who lack an HLA-matched related donor, another option is an alternative donor such as umbilical cord blood, a matched unrelated donor, or a haploidentical donor (including a parent, child, or sibling). Clinical experience with alternative donor HSCT remains limited, and many experts consider HSCT to be an investigational therapy that should only be performed in the context of a clinical trial. This is mainly due to the major and life-threatening toxicities of allogeneic HSCT including acute and chronic GVHD and immunosuppression needed to prevent GVHD." (up to date)

The health plan acted reasonably with sound medical judgment in the best interest of the patient.

The insurer's denial of coverage for outpatient visits for stem cell transplant evaluation and surgery is upheld. Medical Necessity is not substantiated.