Question 52: Was HLH present?
HLH is an abnormal proliferation of macrophages and histiocytes that leads to the phagocytosis of healthy circulating blood cells. Indicate if the patient developed HLH at any time after diagnosis but prior to the start of the preparative regimen; include HLH persisting from diagnosis. If “yes,” continue with question 53. If “no,” continue with question 89.
Question 53: Was the HLH triggered by an acute EBV infection?
HLH may present as an exaggerated response to EBV infection. Epstein-Barr viral infection is ubiquitous and rarely causes life-threatening complications. EBV generally infects the B-lymphocytes; however, in rare circumstances it may infect natural killer (NK) cells and T-lymphocytes. This unusual event is associated with aggressive lymphoproliferative manifestations, including hemophagocytic lymphohistiocytosis (HLH). Indicate if the patient’s HLH was associated with EBV infection. If “yes,” continue with question 63. If “no” or “unknown,” continue with question 54.
Question 54: Was the HLH triggered by any other known condition(s)?
HLH may also present as a response to malignancy or pathogen other than EBV. Indicate if the patient’s HLH was associated with another known condition. If “yes,” continue with question 55. If “no” or “unknown,” continue with question 63.
Questions 55-62: Specify other known condition(s)
Report condition or conditions believed to have triggered HLH. Each of the questions 55-58 and 61 must be answered as “yes” or “no”; do not leave any response blank. If the HLH was in response to a viral infection other than EBV, specify the virus in question 59. If it was a response to a virus not listed, specify in question 60. Questions 59-60 may be repeated to report multiple viral triggers. If the cause of HLH response is best classified as “other,” specify in question 62.
Questions 63-69: Specify site(s) where HLH was present
Indicate “yes” or “no” for each site specified in questions 63-68. Do not leave any response blank. If “yes” is indicated for “other site,” specify the site in question 69. At least one of questions 63-68 must be answered “yes.”
Question 70: Was therapy given for HLH?
Indicate if the recipient received treatment for HLH after the diagnosis and before the start of the preparative regimen. If “yes,” continue with question 71. If “no,” continue with question 89.
Questions 71-72: Date therapy started
Indicate “known” if the therapy start date is documented and specify the first date of therapy in question 72. If the date is unknown, indicate this and continue with question 73.
Questions 73-74: Date therapy stopped
Indicate “known” if the therapy stop date is documented and specify the date therapy stopped using question 74. If the patient received systemic therapy in cycles, specify the first day of the last cycle of systemic therapy. If the patient received a single line or single administration, indicate the last day therapy was administered.
If the date is unknown, indicate this and continue with question 75.
Questions 75-88: Specify therapeutic agents
Allogeneic stem cell transplant is considered the only curative treatment for XLP. However, therapeutic agents may be given in settings where allogeneic HCT is not a viable treatment option or to maintain the patient in the pre-transplant period. Therapy may vary depending on disease presentation. Most therapeutic agents given in the setting of XLP are intended to suppress the patient’s over-responsive, dysfunctional immune system. There is some evidence that cytotoxic therapy, particularly etoposide, may be beneficial in controlling lymphocyte proliferation. B-cell therapy, such as rituximab, may be given to treat EBV infection, since it generally infects B-lymphocytes. Other agents, such as IVIG, may be given to supplement the patient’s immune system and provide artificial immune response.
Indicate “yes” or “no” for each therapeutic agent listed. Do not leave any response blank. If the recipient received an agent that is not listed, check “yes” for “other systemic therapy” and use question 88 to specify the treatment.
Question 89: Did colitis develop?
Colitis refers to inflammation of the large intestine, often manifesting as diarrhea, abdominal pain and bloating, and melena or hematochezia. Indicate if the patient developed colitis at any time after diagnosis but prior to the start of the preparative regimen; include colitis persisting from diagnosis.
Question 90: Did vasculitis develop?
Vasculitis refers to inflammation of the vasculature, including both veins and arteries. Vasculitis may impact blood vessels of any size, from capillaries and arterioles to the great truncal vessels. It is typically caused by autoimmunity. Indicate if the patient had vasculitis (any presentation) at any time after diagnosis but prior to the start of the preparative regimen; include vasculitis persisting from diagnosis. If “yes,” continue with questions 91-94. Answer each of questions 91-93 and do not leave any response blank. If “no” or “unknown,” continue with question 95.
Question 91: Central nervous system
CNS vasculitis refers to inflammation of the vasculature of the brain and/or spinal cord. Indicate if the patient had CNS vasculitis at any time after diagnosis but prior to the start of the preparative regimen; include vasculitis persisting from diagnosis.
Question 92: Pulmonary system
Pulmonary vasculitis involves inflammation of pulmonary vasculature. This can range from the great vessels, such as the pulmonary arteries, to the small alveolar capillaries. Indicate if the patient had pulmonary vasculitis at any time after diagnosis but prior to the start of the preparative regimen; include vasculitis persisting from diagnosis.
Question 93: Other vasculitis involvement
Indicate if the patient had other vasculitis involvement. Specify involvement in question 94. Examples include systemic vasculitis, urticarial vasculitis, or gastrointestinal vasculitis at any time after diagnosis but prior to the start of the preparative regimen; include vasculitis persisting from diagnosis.
Question 95: Did the recipient develop lymphoma?
XLP is associated with a higher incidence of lymphoma, which may be secondary to EBV infection; however, not all lymphomas in the setting of XLP exhibit EBV clonality. There is speculation that lymphoma risk is increased secondary to aberrant iNKT, NK, and T-cell cytotoxic function. The majority of lymphomas seen in XLP patients are T-cell lymphomas. Indicate if the patient had lymphoma at any time after diagnosis but prior to the start of the preparative regimen. If the patient had lymphoma, continue with questions 96-98; also complete Form 2018, Hodgkin and Non-Hodgkin Lymphoma Pre-HCT Data.
Question 96: Was the lymphoma associated with an EBV infection?
Indicate if the patient’s lymphoma was associated with an EBV infection. If the patient’s EBV status was not assessed, indicate “unknown.”
Question 97: Is the tumor EBV positive?
If the patient’s lymphomatous tumor was evaluated for EBV clonality, indicate if it was or was not EBV positive. If the tumor was not evaluated for EBV clonality, indicate “unknown.”
Question 98: Was documentation submitted to the CIBMTR? (e.g., pathology report)
Indicate if a copy of the pathology report and/or immunohistochemistry results are attached. Use the Log of Appended Documents (Form 2800) to attach a copy of the pathology and/or laboratory report(s). Attaching a copy of the report may prevent additional queries.
Question 99: Did the recipient develop hypogammaglobulinemia?
Hypogammaglobulinemia is a condition in which the body does not make enough antibodies or immunoglobulins. It is generally due to decreased numbers of B-lymphocytes. Indicate if the patient had hypogammaglobulinemia at any time after diagnosis but prior to the start of the preparative regimen; include hypogammaglobulinemia persisting from diagnosis.
Question 100: Did the recipient develop aplastic anemia?
Aplastic anemia is a hematologic condition defined by peripheral blood cytopenia(s) and markedly hypocellular marrow with pancytopenia. Indicate if the patient had aplastic anemia at any time after diagnosis but prior to the start of the preparative regimen; include aplastic anemia persisting from diagnosis.
Questions 101-104: Specify therapy given for aplastic anemia
Indicate therapies the recipient received for aplastic anemia after the time of diagnosis and before the start of the preparative regimen. Each of questions 101-103 should be answered as “yes” or “no”; do not leave any response blank. If therapy is best categorized as “other therapy,” specify in question 104.
Section Updates:
| Question Number | Date of Change | Add/Remove/Modify | Description | Reasoning (If applicable) |
|---|---|---|---|---|
| . | . | . | . | . |
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