Copy questions 76-107 to report more than one line of therapy.

Question 75: Was therapy given?

Indicate if the recipient received therapy for HLH between diagnosis and the start of the preparative regimen. If the recipient received therapy, select “yes” and continue with question 76. If the recipient did not receive therapy, select “no” and continue with question 108.

Question 76: Specify the purpose of therapy:

Induction therapies are the initial lines of therapy given to a recipient to bring them into remission. Maintenance therapies are designed to keep the recipients in remission; for some patients with HLH, maintenance is used as an ongoing treatment until a suitable donor can be found for an HCT. If HLH reactivates or relapses following induction therapy or during maintenance therapy, additional therapy is given to treat the reactivation/relapse. Indicate if the line of therapy being reported is “induction,” “maintenance,” or “treatment for disease relapse/reactivation.”

Questions 77-78: Date therapy started:

Indicate if the therapy start date is “known” or “unknown.” If the therapy start date is known, use question 78 to enter the date the recipient began this line of therapy.

Questions 79-90: Specify therapy given:

Systemic treatments vary based on protocol and in many cases are administered in the outpatient setting. A treatment may consist of a single drug or a combination of drugs. Additionally, the drugs may be administered on one day, over consecutive days, or continuously. Indicate “yes” or “no” for each therapy regimen or drug administered for the line of therapy being reported. Do not leave any responses blank. If the recipient received a therapy that is not listed, check “yes” for “other therapy” and specify the treatment in question 90. Report the generic name of the agent, not the brand name.

Question 91: Was this therapy given following the HLH-94/HLH 2004 protocol of the Histiocyte Society?

Indicate if the therapy followed the HLH-94/HLH 2004 protocol of the Histiocyte Society. These protocols are meant to bring the recipient into remission, and then maintain their remission until an HCT can be performed. HLH-94 is a protocol that consists of 8 weeks of induction therapy with Etoposide and Dexamethasone followed by continuation therapy with etoposide, dexamethasone, and cyclosporine until transplant or reactivation of disease. Intrathecal methotrexate may be used in patients who have progressive neurological symptoms or CSF abnormalities. HLH 2004 is a protocol that adds cyclosporine to the induction therapy phase and proceeds similarly to HLH-94.1 For the purposes of this form, the induction and continuation phases of these protocols can be reported as one line of therapy. Prior to transplant, the patient receives a preparative regimen that is separate from these protocols and should be reported on the Form 2000, but should not be included on this form as a line of therapy.

1 Henter JI, Horne A, Aricó M, Egeler RM, Filipovich AH, Imashuku S. Ladisch S, McClain K, Webb D, Winiarski J, Janka G. HLH-2004: Diagnostic and therapeutic guidelines for hemophagocytic lymphohistiocytosis. Pediatr Blood Cancer. V 2007:48(2):124-31.

Question 92: Was CNS disease inactive?

Following this line of therapy, indicate if the recipient’s CNS disease was inactive. Indicate “yes” or “no” and continue with question 93 to report specific response(s). If “unknown,” continue with question 97.

Question 93: Normal or stable CT or MRI of CNS:

Based on CT or MRI of the central nervous system, indicate if previous CNS abnormalities have normalized or stabilized in response to this line of therapy. If the results of the CT or MRI are normal or stable, select “yes.” If there was evidence of new, recurrent, or progressive CNS abnormality, indicate “no.” If the response of CNS abnormalities assessed by CT or MRI is unknown, or if CT or MRI was not performed following the line of therapy, indicate “unknown.”

Question 94: Neopterin level:

Indicate the neopterin level in the cerebrospinal fluid (CSF) following this line of therapy. Indicate “normal” or “elevated.” If an assessment of neopterin levels in the CSF was not done following this line of therapy, select “not done.”

Question 95: Protein level:

Indicate the protein level in the cerebrospinal fluid (CSF) following this line of therapy. Indicate “normal” or “elevated.” If an assessment of protein levels in the CSF was not done following this line of therapy, select “not done.”

Question 96: WBC level:

Indicate the WBC count in the cerebrospinal fluid (CSF) following this line of therapy. Indicate “normal” if there were less than or equal to 5 cells/μL in the CSF. Indicate “elevated” if there were greater than 5 cells/μL in the CSF. If an assessment of WBC count in the CSF was not done following this line of therapy, select “not done.”

Question 97: Was systemic disease inactive?

Indicate if systemic disease consistent with the recipient’s disease was inactive. Assessments of systemic disease include neutrophil count, hemoglobin level, hepatomegaly and splenomegaly evaluation, fibrinogen levels, triglyceride levels, and platelet counts. If the recipient’s systemic disease was inactive following this line of therapy, indicate “yes” and continue with question 98. If the recipient’s systemic disease was not inactive following this line of therapy, continue with 98. If the status of the recipient’s systemic disease was unknown following this line of therapy, select “unknown” and continue with question 105.

Question 98: ANC > 1.0 × 109/L (without growth factor support):

Indicate if the recipient’s absolute neutrophil count (ANC) was greater than 1.0 ×109/L following this line of therapy, without the use of growth factors (e.g., filgrastim). Indicate “yes,” “no,” or “unknown.”

Question 99: Hemoglobin ≥ 9 g/dL without transfusion:

Indicate if the recipient’s hemoglobin level was equal to or greater than 9 g/dL without the use of red blood cell transfusions (within 30 days before test) following this line of therapy. Indicate “yes,” “no,” or “unknown.”

Question 100: Hepatomegaly resolved (≤ 3 cm below costal margin):

Indicate if hepatomegaly was no longer present on physical examination following this line of therapy. Indicate “yes,” “no,” or “unknown.”

Question 101: Normal fibrinogen:

Indicate if the fibrinogen level (factor I; fibrinogen activity; functional fibrinogen; fibrinogen antigen) returned to normal following this line of therapy. Indicate “yes,” “no,” or “unknown.”

Question 102: Normal triglycerides:

Indicate if the triglyceride level has returned to normal following this line of therapy. Indicate “yes,” “no,” or “unknown.”

Question 103: Platelets > 100 × 109/L without transfusion:

Indicate if the recipient’s platelet count was greater than 100 ×109/L without the use of platelet transfusions (within 7 days before test) following this line of therapy. Indicate “yes,” “no,” or “unknown.”

Question 104: Splenomegaly resolved (≤ 3 cm below costal margin):

Indicate if splenomegaly was no longer present on physical examination following this line of therapy. Indicate “yes,” “no,” or “unknown.”

Question 105: Were there any signs of disease relapse/reactivation?

Indicate if there were any signs of relapsed or reactivated disease following this line of therapy. These signs may be present in the central nervous system and detected on radiology (CT/MRI) or clinical neurologic exam, or based on systemic disease assessments. If there were any signs of disease relapse or reactivation following this line of therapy, select “yes” and continue with question 106. If there was no evidence of disease relapse or reactivation following this line of therapy, select “no” and continue with question 108.

Question 106: Specify the date of the relapse / reactivation:

Indicate the date that relapse or reactivation was detected. Use the date of the radiological exam where relapse/reactive was determined, the date of the clinical neurologic exam, or the date the sample was collected for systemic disease assessment.

If the exact date is not known, use the process for reporting partial or unknown dates as described in General Instructions, Guidelines for Completing Forms.

Question 107: Specify the site of the relapse/reactivation:

Indicate if the site of relapse was CNS, systemic, or CNS and systemic. CNS disease is limited to findings in the central nervous system by radiology (CT or MRI) or features specific to the CNS in the clinical neurologic exam. Systemic disease includes findings in the blood counts, triglycerides, ferritin, fibrinogen, and hepatosplenomegaly evaluations.

Last modified: Oct 07, 2020

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